BioQuakes

AP Biology class blog for discussing current research in Biology

Tag: Crispr (Page 1 of 7)

Unlocking the Future: How CRISPR’s New Upgrade is Supercharging Gene Editing

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On April 11, 2025

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Researchers at Yale University have developed a new approach using CRISPR-Cas12a technology to create advanced mouse models for studying genetic interactions that influence immune responses to diseases like cancer. This method allows scientists to simultaneously analyze multiple genes in a single experiment, making genetic research more efficient and insightful. 

File:CRISPR CAS9 technology.png - Wikimedia Commons

Over the past 15 years, advancements in the gene-editing technology CRISPR-Cas9 have provided significant insights into the roles specific genes play in various diseases. However, this technology, which uses a “guide” RNA, a small piece of RNA used in CRISPR gene editing to help scientists find the exact spot on the DNA they want to change, to modify DNA sequences and assess the outcomes, is currently limited to targeting, deleting, replacing, or modifying only single gene sequences at a time. 

 

However, the scientists have now created advanced mouse models using CRISPR  technology that enables them to simultaneously evaluate genetic interactions across various immunological responses to multiple diseases like cancer. Gene editing technologies enable scientists to use enzymes, such as Cas9, as molecular scissors to precisely cut or modify specific segments of DNA or RNA, providing valuable insights into the role of these genes in various disorders. The new tool, CRISPR-Cas12a, allows researchers to simultaneously evaluate the effects of multiple genetic changes that influence different immune system responses, according to the researchers.

 

The research noted this advancement could be valuable in the future to combat a host of pathologies, including cancer, metabolic disease, autoimmune disease, and neurological disorders.

 

This connects to AP Bio in multiple ways. For example, modifying genes can affect cellular functions and processes like cell division and apoptosis. This new technology is used to understand how changes in specific genes can influence cell behavior, which is essential in studying diseases like cancer. In addition, the new developments in CRISPR can be used to study impacts gene editing has on the immune system, and its various responses and functions throughout the body. Lastly, genetic mutation can be better understood through CRISPR editing as it effectively is creating its own “mutation”, changing sequences of codons to form different amino acids after DNA Replication, Transcription, and Translation. What are your thoughts on this research? How can it impact how we know genetics as we understand it today?

Revolutionizing Cancer Treatment: CRISPR’s Role in Classifying BRCA2 Mutations

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On April 11, 2025

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Advancements in gene-editing technologies have significantly enhanced our understanding of genetic mutations associated with cancer risk. A notable study published in Nature (Huang et al. 2025) utilized CRISPR–Cas9-based saturation genome editing to evaluate the functional consequences of all possible single-nucleotide variants in BRCA2 exons 15–26, which encode the DNA-binding domain known for pathogenic missense variants. The researchers introduced nearly all possible single-nucleotide variants into human haploid HAP1 cells and assessed their impact on BRCA2 function. This comprehensive analysis enabled the classification of 6,959 out of 6,960 evaluated variants into seven pathogenicity categories, providing crucial insights for clinical assessments and patient management.

BRCA Genes

BRACA Gene locations by Tessssa13

Similarly, another study published in The American Journal of Human Genetics (Hu et al. 2024) conducted a functional analysis of 462 germline BRCA2 missense variants affecting the DNA-binding domain. This research employed a validated homology-directed DNA repair functional assay to assess the impact of these variants on BRCA2 activity. The findings contributed to the clinical classification of these variants, aiding in the interpretation of genetic test results and informing patient care decisions.

These studies exemplify the transformative potential of CRISPR–Cas9 in functional genomics. By facilitating precise editing and evaluation of genetic variants, CRISPR–Cas9 allows for a deeper understanding of gene function and the consequences of mutations. Such insights are invaluable for developing targeted therapies and personalized medicine approaches. For instance, accurately classifying BRCA2 variants can inform decisions regarding the use of PARP inhibitors, which are particularly effective in patients with specific BRCA mutations.

In our AP Biology curriculum, we’ve explored the mechanisms of DNA repair, including homologous recombination—a process in which BRCA2 plays a pivotal role. Mutations in BRCA2 can disrupt this repair pathway, leading to genomic instability and increased cancer risk. Understanding these molecular mechanisms underscores the significance of precise gene-editing tools like CRISPR–Cas9 in both research and therapeutic contexts.

The application of CRISPR–Cas9 in classifying BRCA2 variants enhances our comprehension of cancer genetics and paves the way for more accurate risk assessments and personalized treatment plans. As research progresses, addressing ethical considerations and ensuring equitable access to these advanced technologies will be crucial.

I chose this topic because someone in my family carries a BRCA1 mutation. When I learned that researchers could now clarify similar variants in BRCA2 using CRISPR, it made me hopeful that one day everyone could know their genuine risk. That kind of clarity can save lives. It also shows how what we learn in class—like transcription, translation, and gene regulation—applies directly to the frontlines of medicine.

I invite anyone who wants to learn more to leave a comment, and I’ll get back to you!

CRISPR Reveals How mRNA Vaccines Work

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On April 8, 2025

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Dr. KIM V. Narry led an important study on mRNA vaccines, like COVID-19, and how cells take them up and respond, as described in the article “Cellular regulator of mRNA vaccine revealed… offering new therapeutic options” from the Institute for Basic Science. This research explains how these vaccines enter cells, carry out their functions, and eventually get degraded. These new insights could lead to the development of better mRNA vaccines and treatments for diseases like cancer and genetic disorders.

In the study, over 19,000 genes were analyzed using a CRISPR-based screen, which led to the discovery of three key factors that affect mRNA vaccine effectiveness. First, the cell surface molecule heparan sulfate (HSPG) was found to help mRNA enter the cell. Second, a protein called V-ATPase was shown to release mRNA inside the cell by creating an acidic environment. Third, the protein TRIM25 acts like a security guard, detecting and destroying foreign mRNA.

The scientists found that proton ions, tiny charged particles, act as signals that tell the cell to launch a defense. When mRNA enters the cytosol, these ions alert TRIM25 to take action. This is the first evidence showing that proton ions can function as immune signals.

Cas9 in complex with sgRNA and target DNA

This article relates to what we’ve learned in AP Biology about protein synthesis and how mRNA is used by ribosomes to make proteins. The study shows that foreign mRNA must avoid being destroyed for this process to happen. TRIM25, part of the innate immune system (which we also studied this year), works to break down foreign RNA, but mRNA vaccines use a modified base (m1Ψ) to protect it and allow translation. This ties into what we’ve just learned about gene mutations. The discovery of proton ions also connects to our unit on cell communication, as it shows how cells respond to threats through chemical signaling.

I found this article fascinating as it included content from almost every unit we have learned this year! Let me know your thoughts in the comments. Did you know a single chemical change in mRNA could make or break a vaccine’s success? Do you think understanding our cells better be the key to curing diseases like cancer?

CRISPR: Bringing back the Woolly Mammoth

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On April 7, 2025

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You may have heard that the gene-editing tool CRISPR has provided hope for treating genetic diseases. Did you know that it is being used to bring back the woolly mammoth?

A company called Colossal Biosciences has been working to revive the species by using genetic modification. They started off by editing five genes in mice, with hopes of one day genetically modifying Asian elephants into woolly mammoths.

The results were cute, fluffy, mice with coats that resemble woolly mammoths. So what steps did they take to create the “mammoth” mice? They first identified mutations in mice that affect fur characteristics, such as curliness, color, and length. They found eight genes linked to these traits, one of which is also disabled in mammoths. Additionally, they identified a mutation in the mammoth genome related to hair pattern and another affecting fat metabolism. Using CRISPR, they disabled five of these genes in fertilized mouse eggs, resulting in 11 pups from 134 edited eggs.

However, much more progress needs to be made. Performing the work on Asian elephants will involve much more effort, and the results may differ significantly. With the size and slow reproduction rate of elephants the experiment will take more time and incur higher costs, but this doesn’t mean it is impossible.

Woolly mammoth (Mammuthus primigenius) - Mauricio Antón

In our AP biology class, we have recently learned about gene expression. Gene expression is accomplished through transcription and translation to create a protein so a trait or phenotype can be expressed. Transcription is the synthesis of RNA using information from DNA, and translation is the synthesis of a protein using information in mRNA. This flow of genetic information from DNA, to RNA, to a protein is known as “Central Dogma”. CRISPR relates to this because it can stop or alter how the gene is expressed, affecting the production of proteins.

It is so interesting to see the advances of CRISPR. I wonder, will it be used to potentially bring back other extinct species? In addition, what are the ethics behind this study? Is it necessary? What else do you think we will see in our future as CRISPR advances?

How far is too far?

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On April 7, 2025

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Colossal Biosciences is working to bring back woolly mammoths by genetically modifying Asian elephants, but progress is slow. They have successfully created mice with mammoth-like fur using CRISPR gene editing, but replicating these changes in elephants will be much harder due to their size, slow reproduction, and complex genetics. So far, only one of the genetic changes in the mice exactly matches the mammoth genome, meaning much more research is needed. Scientists warn that modifying too many genes could cause unintended CAS 4qyzproblems, and some experts argue that creating a furry elephant is not the same as bringing back a true mammoth. Ethical concerns and practical challenges also raise doubts about whether this project will succeed.

The idea of bringing back woolly mammoths with the help of gene editing has caused controversy since everyone has a different perspective on this.  Colossal Biosciences, is a company that is interested in doing do with woolly mammoths as they successfully created mice with fur similar to that of a mammoth. It is hard to succeed in this mission due to over 1.5 million differences within genes of a mammoth compared to an Asian elephant.

A recent study performed by Colossal showed that eight genes affected characteristics associated with fur such as color, length, etc. Scientists used CRISPR gene editing to turn off five specific genes in mice, which ultimately led to fur that is similar to that of a mammoth. Although this is progress, it is hard to also find similarities in elephants to show cross over. Mice are very small and more simple, whereas elephants are very large and have long reproducing cycles.

Additionally, many researchers recognize that extensive genetic modifications can be very complicated and come with some risks. If you change too many genes it can result in problems with their metabolism and lead to problems within the organism’s body.

Another question and complexity within gene editing is if it is ethical. Many people, specifically researchers believe that it is very invasive especially in certain situations regarding the eggs of an organism.  Another argument that is made is that even if an elephant is successfully modified to have fur, it would still be an elephant, not a mammoth.

This raises curiosity if you can make a species not extinct which seems impossible, or if you can just use gene editing to make modern hybrids. This data and research portrays that scientists can attempt and succeed in replicating specific traits, but overall it is extremely difficult and complicated to just bring a species back. This shows that while science can replicate certain traits, bringing back a long-extinct species is far more complex than just altering a few genes. Advancements and new studies  in genetic engineering raise many questions if it is moral as humans.

This connects to what we have learned in AP Biology since it connects to DNA. DNA is shown in all living things since it provides a set of instructions from the parents. In regards to the woolly mammoth, scientists studied the DNA of the mammoth to find genes such as fur. They then used CRISPR gene editing and they changed the DNA of mice by turning off or just modifying certain genes to make their fur  look like a mammoth. Changing the DNA led to a different appearance since it changed their features. So, ultimately the DNA is changed to make the mice have a similar appearance to the woolly mammoth. I wonder if majority of people think this is ethical or unethical to the organism, and if so, how far will CRISPR go?

CRISPR: Zebrafish and gRNA

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On April 7, 2025

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CRISPR-Cas9 is a revolutionary gene-editing technology, first invented by biochemist Jennifer Doudna and microbiologist Emmanuelle Charpentier. This method, aptly dubbed “genetic scissors,” enables scientists to cut DNA through precise removals and alterations of DNA sequences, which allows for the correction of genetic errors and the manipulation of gene expression. This technology has extreme potential to correct mistakes in DNA that cause conditions ranging from congenital birth defects and cancer. Additionally, CRISPR utilizes gRNAs (guide RNAs) to target specific sections of DNA/RNA in a cell. These gRNAs enable scientists to use CRISPR technology to cut genetic information with high precision.

Last month, researchers from Spain and the USA worked together to test how well modified CRISPR-RfxCas13d gRNAs could improve gene targeting in zebrafish embryos. They wanted to see how they could further improve CRISPR technology to target specific sequences in living organisms. They used chemically-modified gRNAs along with a protein called RfxCas13d to turn off genes active during the developmental stages in zebrafish embryos. They then modified the protein to be particularly efficient in zebrafish cells, and compared their various results of the CRISPR targeting.

Zebrafish (26436913602)

The scientists found that chemically-modified gRNAs do in fact improve the process of cutting out genetic information in living embryos. They further proposed that this process could be replicated to enhance the effectiveness of CRISPR editing in other organisms, too.

In this past AP Biology unit, we have learned about how harmful mutations can be, and what kinds of disorders can arise from those mutations. In class, we learned the different types of mutations (substitution, deletion, etc) and how even one seemingly small change in genetic code could have devastating effects on the affected organism; CRISPR might eventually be used to correct these harmful mutations in humans. Although there is still much more research to be done on CRISPR technology to reach the point at which we can begin to resolve genetic problems in humans, the possible uses of CRISPR are vast.

Whenever I think of CRISPR, I’m always reminded of the novel Brave New World, by Aldous Huxley. The book is set in a dystopian world where embryos are genetically modified to create people that “perfectly” fit their specific hierarchical positions and roles in society. As a result of gene editing, people in the depicted society lack true emotions and individuality. Although Huxley’s novel was written decades before CRISPR was invented, its warning about the dangers of manipulating genes rings true, especially now. What do you think of the ethical dilemma that CRISPR gene editing poses? How far is too far when it comes to scientific and technological progress, and who gets to decide those limitations?

Using CRISPR and AAV gene insertion to cure disease

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On April 7, 2025

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What is CRISPR?

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) are genes that quickly repeat DNA base sequences that are mechanically edited using a protein called Cas9. This system is based on the Cas9 protein’s ability to splice a given DNA sequence by being given a matching RNA code.  This is incredibly similar to how alternative RNA splicing works in gene regulation for eukaryotic organisms.  In the same way that alternative RNA splicing removes sequences of RNA code during the RNA processing stage of gene expression to create a variety of different proteins, the Cas9 protein removes sequences of code from DNA to synthesize unique proteins. When given a specific RNA sequence, the Cas9 protein will match that RNA sequence to a sequence of DNA, commencing the splicing of the DNA at that location. This process was discovered by Emmanuel Charpentier et al. in bacteria that use the Cas9 protein to target harmful DNA code inserted into the bacterial cytoplasm by viral organisms. Using the Cas9 protein, genetic engineers are able to remove DNA sequences in organisms to cause them to create different proteins and show a wanted phenotypic trait.

DNA alternative splicing

What is AAV?

AAV is a method of inserting DNA into a non-embryonic organism through structures similar to that of viruses.  However, instead of containing harmful virus DNA, these vessels contain modified DNA using CRISPR to force a desired gene expression at a given location in the human body.  AAV-based delivery systems are able to change a person’s genome during their lifetimes, potentially reducing the harm of/eliminating genetic diseases.

Experimentation

Dr. Peace Chinonyerem Ike et al. ran an experiment this year on pediatric males to test the effectiveness of using CRISPR editing through AAV to alter the genomes of certain cells. The diseases they chose are those that have been linked to the X-linked chromosome, meaning that men are more likely to get these diseases such as hemophilia and DMD. This is because X-linked traits in men come exclusively from the mother making it statistically more likely that they will express the phenotype of that gene. After experimentation over five years with treating patients, they saw that using CRISPR was significantly more effective than previous methods for reducing symptoms in these x-linked traits (such experiments include ZFNS).

What Does This Mean

After concluding that CRISPR can be used to treat genetic diseases on the X chromosome we may be able to expand its capabilities to more genetic illnesses such as Cystic Fibrosis that are autosomal. Using CRISPR, we can move medicine away from preventing side effects to outright curing illnesses at the level of the genome. This is a huge feat in the field of medicine and may predict a wave of new treatment strategies involving DNA modification in the future. I believe that, although this possible form of treatment is monumental, we must be incredibly cautious about using AAV-based systems as they may cause harmful mutations in the person’s genome. If we do not make sure that this treatment is safe we may cause multiple people to undergo harmful gene mutations and, for example, develop cancer. So you think that we will see the application of this treatment form in practice? If so, what do you think the ethical and moral ramifications are when using gene editing on a developed person?

CREME: Revolutionizing Gene Expression Analysis for Drug Discovery

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On April 7, 2025

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DNA / Naturalis Leiden

CREME is an AI-powered virtual based on CRISPRi, developed by Cold Spring Harbor Lab Assistant Professor Peter Koo and his team, allowing geneticists to run thousands of experiments simply with the click of a button. 

To understand CREME, we first need to understand CRISPR and CRISPRi (CRISPR interference). CRISPR uses a protein called Cas9 to cut certain DNA out of a genome, similar to how introns are cut out in RNA processing. CRISPRi, on the other hand, uses a modified Cas9 called dCas9 not to cut out certain DNA sequences but to block them during RNA transcription, acting similarly to a repressor. During transcription, CRISPRi can interfere with RNA Polymerase during transcription initiation or transcription elongation.

CREME is, in a sense, an AI-powered virtual CRISPRi. This is a major advantage to CRISPRi because, as Koo explains, “CRISPRi is incredibly challenging to perform in the laboratory,” further saying that “the number of perturbations and the scale” also limit CRISPRi in the laboratory. 

However, by using CREME, the aforementioned limitations seemingly disappear, as it becomes possible to do “hundreds of thousands of perturbation experiments.”

Koo and his team also demonstrated that CREME could be used to help discover genetic rules. To do this they tested CREME on another AI-powered genome analysis tool called Enformer. Enformer is good at making accurate predictions on gene expression, but we don’t know what rules of gene regulation Enformer or other models like Enformer are basing their predictions on.

With CREME, Koo and his team found several rules that Enformer based its predictions off of. These findings are compelling in the possibilities in drug discovery. By understanding how different genes are expressed, drugs can be more easily discovered.

Do you have any ideas on how changing gene expression can impact public health?

Beyond Genetic Scissors: How CRISPR-Cas12a Is Editing the Future of Medicine

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On April 7, 2025

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Cas12a (AsSpf1) in complex with crRNA and target DNA

Imagine if your DNA came with a backspace key — thanks to CRISPR, it kind of does. This idea may seem crazy, and it is, but let me explain. CRISPR is a unique technology that allows scientists to edit parts of the genome by removing, adding or altering sections of the DNA sequence. How does it do this? Well it’s a little complicated. The CRISPR-Cas9 system has two fundamental molecules, the enzyme Cas9 and a piece of RNA called guide RNA (gRNA). gRNA is a small piece of pre designed RNA that contains bases complementary to a specific DNA sequence. This means that the gRNA will only bind to the targeted sequence and will “guide” the Cas9 to the right part of the genome. Cas 9 is playfully referred to as “genetic scissors” as it makes precise cuts in both strands of the DNA at the targeted spot in the genome. When the cell recognizes its DNA is damaged it activates repair mechanisms, allowing scientists to swoop in and insert, delete, or modify genetic material at that site.

Seems amazing right? The one catch is that CRISPR can only target, delete, replace, or modify one gene sequence at a time using a single guide RNA, making it difficult to study multiple genetic changes at once. However, a brand new study at Yale school of medicine has developed a new technologie that can help researchers simultaneously assess the impact of multiple genetic changes involved in a variety of immune system responses. This new tool is called CRISPR-Cas12a, and seems to have huge implications. Sidi Chen, an associate professor of genetics and neurosurgery at Yale School of Medicine and a pioneer in the field of CRISPR technology, led the lab in making four specially engineered mouse lines that allowed the scientists to study “complex genetic interactions and their effects involved in many disorders.”

Using Cas12a Chen’s lab was able to induce and monitor changes in the immune cell in response to gene editing, fine tuning sets of genes in different directions at the same time. The researchers were also able to generate rapid production of new disease and treatment models, including genetic disease in the liver, lung cancer, and skin cancer.

This all represents a huge step forward in the world of CRISPR gene editing, as according to Chen these developments will be a powerful tool for scientists developing new treatments for a wide range of diseases, from cancer and metabolic disorders to autoimmune and neurological conditions.

In fact CRISPR treatment has already been utilized with real patients. A clinical trial launched by the University of Pennsylvania in 2019 used CRISPR to engineer T cells that could better detect and attack cancer cells. The treatment entailed removing three genes that interfered with the immune system’s response to cancer and adding one gene that helped the T cells recognize cancer cells. While the trial focused on safety and the results were modest, the trial proved that CRISPR treatment can be done in a safe and meaningful way. This coupled with the work done in Chens Lab at Yale lays the groundwork for groundbreaking discoveries in the future. 

As a student of AP bio the work being done in the world of CRISPR gene editing is especially interesting as we recently just finished our unit on molecular genetics which centered around DNA, RNA, and Gene Regulation/Expression, topics that are very relevant to CRISPR technology. Having this background made me more curious as I researched the topic. For example we learned in class that before DNA can be transcribed, helicase needs to “unzip” the DNA, which made me wonder what unzips the DNA so the gRNA can bind to the complementary bases? Does Cas9 unwind the DNA due to its job as the “genetic scissors”? Or is helicase itself involved?

Another question I had connected to what I learned in AP bio was about frame shifts. If CRISPR cuts targeted spots in the genome, then how do researchers prevent frame shifts that could cause totally different proteins to be produced? 

If you know the answer to any of these questions please let me know in the comments!

How CRISPR is Changing the Way We Treat Illness

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On April 7, 2025

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Introduction: Imagine a world where genetic diseases like sickle cell disease (SCD) are no longer a life sentence. Recent advancements in gene editing, particularly using CRISPR technology, are turning this vision into reality. As an AP Biology student fascinated by genetics, I was captivated by a groundbreaking therapy that utilizes CRISPR to treat SCD, a condition we’ve explored in our studies on genetic mutations and their physiological impacts.

 

Summary of Main Research Article:

 

A recent article titled “Cutting Edge Gene Therapy” discusses the development and approval of Cassava, a CRISPR-based gene therapy for individuals suffering from sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Researchers discovered that higher levels of fetal hemoglobin (HbF), which has a higher oxygen affinity than adult hemoglobin, can alleviate symptoms of these diseases. Casgevy works by editing blood-forming stem cells to promote HbF production, thereby reducing the impact of SCD and TDT. Clinical trials indicated that 93.5% of SCD patients did not experience pain crises for at least a year post-infusion. Notably, this therapy became the first CRISPR-based treatment to obtain FDA approval in December 2023. 

Orginal Article: https://time.com/7094725/vertex-pharmaceuticals-and-crispr-therapeutics-casgevy/

Additional Resources:

 

  1. Innovative Genomics Institute: CRISPR Clinical Trials Update https://innovativegenomics.org/news/crispr-clinical-trials-2024/

 Provides an overview of ongoing CRISPR clinical trials and their implications.

 

  1. Nature: CRISPR genome-editing grows up https://www.nature.com/articles/d41586-024-04102-w

 Discusses the maturation of gene-editing technologies for treating various disorders.

 

Connection to AP Biology:

 

In our AP Biology curriculum, we’ve studied the central dogma of molecular biology, which describes the flow of genetic information from DNA to RNA to protein. Sickle cell disease is caused by a point mutation in the β-globin gene, leading to the production of abnormal hemoglobin S. This mutation results in red blood cells adopting a sickle shape, causing blockages in blood vessels and leading to pain and organ damage. The CRISPR-based therapy directly relates to our discussions on gene expression and mutation impacts, as it involves editing the genome to reactivate the production of fetal hemoglobin (HbF), which can compensate for the defective adult hemoglobin. This therapeutic approach exemplifies the practical applications of our understanding of gene regulation and expression.

 

Personal Reflection:

 

The potential of CRISPR to cure genetic diseases is both exciting and thought-provoking. Learning about Chevys success in treating SCD patients without pain crises for extended periods is inspiring. However, it also raises questions about accessibility and long-term effects. How can we ensure that such groundbreaking treatments are available to all who need them? What ethical considerations should guide the application of gene editing in humans? I’d love to hear your thoughts on these issues.

 

Tags: CRISPR, gene editing, sickle cell disease, gene therapy, fetal hemoglobin

 

Images:

< Diagram explaining steps behind simple CRISPR-based diagnostics

 

This figure shows how CRISPR can be used for quick diagnostics in addition to gene editing. Scientists can develop extremely sensitive, field-deployable diagnostic assays by employing CRISPR proteins to identify particular viral or bacterial DNA sequences. Even though I wrote a piece about gene therapy, this demonstrates how CRISPR technology has many uses outside of medicine and is changing the way we identify illness.

 

 

 

CAS 4qyz

 

The Cas9 protein, the molecular “scissors” in CRISPR systems, is depicted in this 3D model. Cas9 finds and cuts specific DNA sequences under the guidance of RNA, allowing for accurate modifications. The physical complexity of this protein reminds me that even the tools we use to rewrite DNA are themselves the result of evolution — making CRISPR a case of biology editing biology.

 

CRISPR-Cas9

 

This picture explains the CRISPR-Cas9 mechanism: a guide RNA (gRNA) directs the Cas9 protein to a particular DNA sequence, where it chops the strand. After that, the cell fixes the damage, either causing the gene to malfunction or enabling researchers to introduce a corrected form. It helps connect what we study in class with actual medical advancements and effectively illustrates the mechanism underlying treatments like Casgevy.

 

How CRISPR-Cas12a is Changing Medical Research

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On April 7, 2025

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Gene editing technology has evolved dramatically over the past decade and the latest advancement from Yale University is set to revolutionize how we approach disease modeling and genetic research. The newly developed CRISPR-Cas12a tool enhances the capabilities of the traditional CRISPR-Cas9 system, offering the ability to assess the impact of multiple genetic changes simultaneously. This innovation could greatly accelerate medical research and therapeutic developments.

The schematic diagram of CRISPR-Cas9

The traditional CRISPR-Cas9 system, which has been a fundamental tool in genetic engineering, uses a guide RNA to target and modify specific DNA sequences. In the CRISPR system, “spacer” sequences are transcribed into short RNA sequences known as CRISPR RNAs that guide the system to specific DNA sequences. Once the target DNA is located, the Cas9 enzyme, produced by the CRISPR system, binds to the DNA and cuts it, effectively silencing the targeted gene. Alternatively, by using modified versions of Cas9, researchers can activate gene expression instead of cutting the DNA. These methods allow researchers to study the gene’s function.

In AP Biology, we recently learned about transcription, gene expression, and gene regulation. Just as transcription factors in eukaryotic cells help initiate and regulate the transcription of DNA into RNA, the CRISPR system uses crRNAs to identify and modify specific DNA sequences, showcasing a direct application of these biological principles in advanced genetic engineering. Both transcription factors and CRISPR systems achieve their targeted function by recognizing specific DNA sequences through RNA interaction.

However, CRISPR/Cas9’s application is somewhat limited, as it can only modify one gene sequence at a time. Also, when CRISPR/Cas9 is applied directly to embryos, it’s not possible to selectively choose the genetic changes, making it difficult to ensure the desired modifications are made without unwanted mutations. The recent breakthrough by Yale’s research team has culminated in the development of CRISPR-Cas12a, which can target multiple genes at once and assess their collective interactions in disease processes. This capability to edit multiple genes simultaneously enhances the effectiveness of “Crispants,” a method for rapidly knocking out genes by targeting several loci at once in the F0 generation of animals. This technique significantly accelerates early validation in preclinical studies, providing a quicker way to evaluate therapeutic hypotheses.

In our AP Biology course, we learned that the F0 generation refers to the first generation of offspring in a genetic study, which is crucial for observing initial traits and mutations.

Yale researchers have used this technology to develop advanced mouse models that mimic human immune responses more accurately, allowing for a better understanding of complex genetic interactions in diseases like lung cancer, skin cancer, and various metabolic and autoimmune diseases.

I find it interesting that new advances are being made that will hopefully lead to improvements in healthcare treatments in the future. Personally, I was diagnosed with autoimmune hepatitis this year, and my mom has hashimotos. I know its very common for women to get these autoimmune diseases and I hope the future of science and healthcare will allow for earlier detection, prevention, and care.

Have you heard about CRISPR before this blog post? Are you interested to see the advancements that can be made in the future and how they may impact you and future generations?

CRISPR Gene Editing can have Unwanted Effects

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On April 7, 2025

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A group of researchers at the University of Zurich have completed a study to test whether CRISPR gene editing technology can be used to correct the DNA sequence error in people with chronic granulomatous, a rare genetic disease that causes white blood cells to be unable to kill certain pathogens. They found that the technology was successfully able to insert the two nitrogenous bases, that are missing in people with this disease, into the DNA sequence. While this process was successful, they also found that, at the same time, it caused further damage to the DNA. 

After performing the gene editing process, the scientists found new defects in the newly repaired cells. In certain cells, the entire section of the DNA where the editing took place was missing. This is due to the NCF1 gene and its characteristics. It shows up three times in the DNA; once as an active gene, and twice as pseudogenes, which are sections of DNA that resemble active genes but are in fact nonfunctional. The CRISPR technology cannot distinguish between the two versions of the gene and therefore ends up editing multiple portions of the DNA, resulting in gene segments being misaligned or missing. The consequences of such errors are still not fully understood and can be unpredictable. Therefore medical professionals are hesitant to use this method of treatment for chronic granulomatous disease. Other research teams have continued this work to find a safer method of treatment using CRISPR. Do you think that CRISPR should be used to cure diseases like chronic granulomatous despite possible risks? 

DNA Structure+Key+Labelled.pn NoBB

Model of DNA Structure

This connects to the AP Biology subjects of DNA structure and function. The CRISPR technology performs edits to DNA sequences such as adding or removing certain nitrogenous bases, which is closely related to DNA structure. It also relates to DNA function as changing the sequence of the DNA will alter what the DNA codes for. This changes how certain portions of DNA affect cell processes by, for example, producing different proteins.

New CRISPR Technology Can ‘Press Pause’ on Specific Genes

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On April 7, 2025

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Scientists have discovered a new version of CRISPR that is reversible rather than permanent.

This new genetic tool is known as the IV-A CRISPR system. Traditional CRISPR technology works by producing short RNA strands that help the system locate matching DNA sequences. Then, when the target DNA is identified, the Cas9 enzyme binds to it and cuts, disabling the gene. IV-A CRISPR disables the gene without cutting the DNA by continuously influencing it. This system temporarily turns off genes, offering greater control. For example, researchers could turn off a gene and later turn it back on to see the effect it has.

The schematic diagram of CRISPR-Cas9

Diagram of Cas9 enzyme

Looking ahead, Pausch and his team plan to study how CRISPR molecules change while silencing genes, then explore medical applications. The system could enable precise genome editing, temporary gene expression control, or modify epigenetics, changes in gene function that do not involve changing the DNA sequence. Further research and discovery could lead to huge benefits for both medicine and agriculture. I think that it is amazing the range of possibilities that this technology could have for society but I also wonder about potential controversies that could arise. What do you think? If you have any thoughts that you would like to share please write them in the comments!

This relates to the AP biology topic of genetics. It relates to what we have learned about RNA and DNA. mRNA is made from DNA through transcription and then it is used to make proteins. For CRISPR, they use another kind of RNA in a completely different way. They use it to find a matching DNA sequence rather than code for a protein. It also relates to what we have learned about gene regulation. Both traditional CRISPR technology and IV-A CRISPR are essentially repressing for the gene by influencing the DNA, impacting transcription.

CRISPR: Gene Editing and Its Ethical Dilemmas

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On March 26, 2025

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CRISPR: Revolutionizing Gene Editing and Its Ethical Implications

The revolutionary technique known as CRISPR-Cas9 has revolutionized the study of genetics in recent years. With the use of this potent instrument, researchers can precisely alter the DNA of living things, creating new opportunities in fields such as agriculture and medicine. But great power also comes with great responsibility, and the ethical issues surrounding CRISPR are just as important as its possible uses.

Cas9 in complex with sgRNA and target DNA

Understanding CRISPR-Cas9

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a natural defense mechanism found in bacteria. These organisms use CRISPR sequences to identify and cut the DNA of invading viruses, thereby neutralizing threats. Scientists have harnessed this system, pairing it with the Cas9 enzyme to create a method for editing genes with unprecedented accuracy. The process involves designing a small piece of RNA that matches the target DNA sequence, guiding Cas9 to the exact location for modification.

 

Applications in Medicine

Treating hereditary illnesses is one of the most exciting uses of CRISPR. Scientists are looking at using it to fix the mutations that cause diseases like cystic fibrosis and sickle cell anemia. Instead of just treating symptoms, CRISPR may be able to treat diseases at their root by fixing damaged genes. The safety and effectiveness of these methods are being evaluated in clinical studies, giving patients with diseases that were previously incurable hope.

 

Advancements in Agriculture 

CRISPR is causing a stir in agriculture in addition to medicine. Crops that are more resilient to environmental stressors, illnesses, and pests are being developed by scientists. To solve food security issues in different regions of the world, gene-edited plants, for example, can be designed to have improved nutritional profiles or to tolerate drought conditions. These developments may result in increased crop yields and more environmentally friendly farming methods.

Moral Aspects to Take into Account

Although CRISPR has enormous potential advantages, there are also serious ethical issues. The potential for off-target effects, in which unwanted portions of the genome are changed and result in unanticipated outcomes, is one significant problem.

Cas9 in complex with sgRNA and target DNA A digital sketch depicting a gene-edit

AP Bio Relation

We have studied the mechanisms of transcription, translation, and DNA replication in our AP Biology course. Gaining an understanding of these basic mechanisms is essential to understanding how CRISPR-Cas9 can accurately target and alter particular genes. This technology shows how fundamental knowledge may result in game-changing inventions and is an example of how molecular biology topics we have studied can be applied in real-world settings.The discovery of CRISPR-Cas9 marks a turning point in science by combining enormous promise with difficult moral dilemmas. What do you think about the application of gene-editing technology to people? How does society strike a balance between moral obligation and innovation? Leave a comment below with your opinions.

 

CRISPR to the Rescue: Tackling Cystic Fibrosis at the Genetic Level

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On March 7, 2025

In Student Post

Cystic fibrosis (CF) is a genetic disorder that affects the lungs, pancreas, and other organs, leading to severe respiratory and digestive issues. CF is caused by mutations in the CFTR gene, which codes for a protein responsible for regulating salt and water movement in and out of cells.

Cystic Fibrosis Symptoms

As we learned in AP Biology, mutations in a single gene can lead to significant changes in the structure and function of the resulting protein, which then disrupts cellular processes. Specifically, the mutation in the CFTR gene alters the primary structure of the CFTR protein, which leads to improper folding. This improper folding prevents the protein from reaching the cell membrane where it’s needed, impairing its ability to transport chloride ions. This disruption in protein structure and function results in the thick mucus buildup that characterizes CF. This directly connects to the concept of protein structure-function relationships that we studied, where even small changes in the amino acid sequence can have profound effects on the protein’s overall shape and functionality.

Traditional treatments for CF focus on managing symptoms, such as using medications to thin mucus or conducting lung therapies. While these treatments improve quality of life, they do not cure the underlying genetic cause of the disease. However, recent advances in genetic editing are offering hope for a permanent cure. CRISPR-Cas9, a revolutionary gene-editing tool, has made it possible to target and correct the specific mutation in the CFTR gene that causes cystic fibrosis.

In recent studies, researchers have used CRISPR-Cas9 to edit the CFTR gene in cultured human cells and animal models. This process involves precisely cutting the DNA at the site of the mutation and either repairing the defective gene or inserting the correct version of the gene. By correcting this genetic flaw, scientists hope to restore the proper function of the CFTR protein, alleviating the symptoms of cystic fibrosis. The process also involves delivering the CRISPR tool into cells using viral vectors or nanoparticles, which can be challenging but has shown promising results in preclinical trials.

One groundbreaking clinical trial conducted on CF patients involved modifying their lung cells using CRISPR. During this trial, scientists extracted cells from patients’ airways, edited them with CRISPR to correct the CFTR mutation, and then reinfused them into the patients. The results were encouraging, as many patients showed an improvement in lung function and a reduction in symptoms. The ability to edit the CFTR gene directly in the body is a significant step forward and opens up new possibilities for treating not just CF, but other genetic disorders caused by similar mutations.

This trial also revealed important insights into the challenges of gene editing in humans. While the results were promising, there were concerns about the precision and long-term effects of CRISPR. Some patients experienced temporary side effects, such as mild inflammation, but the treatment itself showed minimal adverse reactions. Researchers are continuing to monitor these patients to ensure the safety and long-term efficacy of the procedure.

In conclusion, CRISPR technology has the potential to revolutionize the treatment of genetic diseases like cystic fibrosis. By directly targeting the cause of the disease at the genetic level, CRISPR could offer a permanent cure, eliminating the need for lifelong symptom management. This success could pave the way for future gene-editing treatments for a variety of genetic disorders, such as Duchenne muscular dystrophy or even sickle cell disease, showing how gene editing could be the next frontier in personalized medicine.

I’m excited about the promise of CRISPR in treating cystic fibrosis because it offers real hope for patients who have long suffered from the debilitating symptoms of this disease. I chose to write about this topic because I recently watched the movie 5 Feet Apart, which inspired me to learn more about cystic fibrosis and how scientific advancements like CRISPR might change the future of treatment for these patients.

What are your thoughts on CRISPR and its potential to treat cystic fibrosis and other genetic diseases? Do you think we’ll see more gene-editing therapies become available in the near future? I’d love to hear your opinions in the comments!

CRISPR: Rewriting the Script in Cancer Treatment

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On January 20, 2025

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Cancer continues to impact millions of people each year around the world; however, new breakthroughs in cancer treatment using CRISPR technology are set to transform how we can combat this complex disease. By leveraging CRISPR’s gene-editing capabilities, researchers are unlocking new possibilities to enhance immune responses, optimize therapies, and develop more precise and effective treatments.

A groundbreaking study from Harvard Medical School, led by LaFleur et al. and Milling et al., explored how CRISPR can reprogram T cells to more effectively fight cancer. Cancer cells typically evade the immune system by downregulating antigen presentation or suppressing the immune system. The researchers addressed these challenges by targeting specific genes in T cells to enhance target recognition(improved the T cells’ ability to recognize tumor antigens), increase the length of the immune response, and strengthen activation(amplifying the response on the detection of cancer). These genetically modified T cells show significant improvements in combating cancer in laboratory and preclinical models than the typical T cell. This showcases their potential to develop more effective immunotherapies, especially for cancers resistant to traditional treatments.

CAR T-Cell immunotherapy diagram by (OHC CAR-T team)

Another innovative study by Lei et al. explored CRISPR’s role in enhancing CAR(Chimeric Antigen Receptor) T-cell therapy, a promising approach that modifies a patient’s T cells to target cancer cells. While CAR T-cell therapy has shown success, it faces challenges like limited efficacy against solid tumors, safety concerns, and high costs. CRISPR offers potential solutions by improving efficacy(Enhances T-cell function and persistence through precise gene edits), enhancing safety(disables genes responsible for adverse effects like cytokine release syndrome), and reducing costs(streamlining the manufacturing process to make the therapy more accessible)

This study emphasizes how CRISPR can address existing barriers, making CAR T-cell therapy safer, more effective, and available to more patients.

The potential of CRISPR extends beyond these studies. CRISPR has opened up a new frontier in cancer research by directly editing the genes within cancer cells, disrupting oncogenes, and reshaping immune responses. For example, recent findings revealed that CRISPR can target oncogenes in leukemia cells, reducing their ability to proliferate and making them more vulnerable to existing treatments (Carlo et al.).

These advancements directly connect to what we’ve studied in AP Biology. The role of CRISPR in gene editing demonstrates the power of manipulating transcription and translation, concepts which are directly related to nucleic acids. Additionally, the focus on T-cell activation and immune responses ties into our understanding of cell communication and the immune system’s intricate pathways.

I chose to write about this topic because it represents a deeply personal and hopeful turning point in cancer treatment. My mom battled breast cancer, and seeing her fight the disease made me acutely aware of the challenges cancer patients and their families face.

What do you think about CRISPR’s role in transforming cancer treatment? Could we one day eliminate certain types of cancer altogether? Share your thoughts and let’s discuss!

Cracking the Code of CRISPR: A Novel Approach to Genetic Editing

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On November 7, 2024

In Student Post

NHGRI-97218

The latest breakthroughs in CRISPR-Cas9 technology are opening doors to treating genetic disorders in groundbreaking ways. CRISPR, a precise gene-editing tool, has transformed how scientists approach genetic research by offering a way to correct DNA errors directly. The potential of CRISPR to correct genetic mutations that lead to diseases like sickle cell anemia, muscular dystrophy, and cystic fibrosis is a beacon of hope in genetics.

CRISPR involves using a guide RNA to lead the Cas9 protein to a specific DNA sequence, making a precise cut and replacing faulty genes with healthy ones. This precise targeting is possible because of complementary base pairing, a concept we studied in Unit 1. Just as we learned that adenine pairs with thymine and cytosine with guanine, CRISPR relies on this base-pairing rule to locate the exact spot on the DNA where editing is needed.

CRISPR-Cas9 mode of action

 

Research is ongoing, and human clinical trials are actively testing CRISPR’s potential. According to a recent article in Scientific American, an advancement in CRISPR called “base editing” allows scientists to make even more precise changes at the molecular level. This improvement holds promise but raises concerns about “off-target effects,” or unintended DNA modifications that could lead to unpredictable consequences. Keeping abreast of these developments is crucial for understanding the full implications of CRISPR technology.

In AP Biology, we are currently studying Cell Communication in Unit 3, which explores how cells interact and respond to signals. CRISPR’s potential to edit genes involved in signaling pathways could allow scientists to study how changes in specific genes alter cell communication. For instance, modifying genes related to cell growth could provide insights into conditions like cancer, where cell signaling often goes awry. This means that CRISPR could treat diseases and help us understand how they develop and progress.

 

 

Another connection to our AP Bio studies lies in our understanding of biomolecules. In Unit 1, we learned about macromolecules like proteins and nucleic acids. CRISPR is a real-world example of how proteins (like the Cas9 enzyme) interact with nucleic acids (DNA) to create targeted genetic changes. The Cas9 protein is engineered to recognize specific DNA sequences, allowing precise alterations at the molecular level.

I am genuinely excited about CRISPR’s potential to cure genetic disorders once thought incurable as the idea that we may one day be able to “edit” our DNA is incredible. However, it is important to note that this excitement is tempered by some ethical concerns. With the possibility of “designer babies” and unforeseen side effects, should there be limits on how CRISPR is used in humans? As we move closer to learning about genetics and inheritance in AP Biology, I wonder: Where should we draw the line between healing and enhancement? I would love to hear your thoughts on this!

Unlocking a Cure: CRISPR Takes on Sickle Cell Disease

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On November 5, 2024

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Sickle cell disease (SCD) and beta-thalassemia are both caused by mutations in the hemoglobin gene, and can lead to severe anemia. As we learned in AP Biology this year, a single mutation in the amino acid sequence of a protein can have profound effects on its structure and function. In the case of sickle-cell hemoglobin, the mutation involves a change from glutamic acid, a polar amino acid, to valine, a non polar amino acid at the sixth position of the betaglobin chain. This substitution alters the primary structure of the hemoglobin protein and leads to significant changes in its tertiary structure. The introduction of the hydrophobic valine creates an exposed region on the surface of the protein, which promotes aggregation of hemoglobin molecules. These aggregates distort the red blood cells into a sickle shape, causing blockages in blood flow and leading to various health complications. This demonstrates how a single point mutation can disrupt the delicate balance of  a protein’s three-dimensional shape, ultimately affecting its entire functionality.

Normal and sickle red cells

Treatments for these diseases typically require regular blood transfusions, which is not only inconvenient and disruptful for the daily life of patients, but also creates risks such as infections. But recently, researchers have been able to use the gene editing tool of CRISPR-Cas9 to specifically modify patients’ stem cells (specifically the cells that increase blood cells). While modifying, doctors can reactivate fetal hemoglobin production in the body, which normally goes away after birth. Fetal hemoglobin can moderate sickle cell disease through increased oxygen saturation. This process involves taking hematopoietic stem cells (HSC) from either the patient’s bone marrow or blood, and then using CRISPR-Cas9 to edit specific genomes. With this treatment, increased levels of this fetal hemoglobin replace the defective hemoglobin that exists in an adult patient and ultimately alleviate symptoms of both SCD and beta-thalassemia.

This article also discusses specific clinical trials where several patients were enrolled who had a confirmed diagnosis of SCD or beta-thalassemia. During these trials, patients went through a procedure, leukapheresis, to extract their HSCs. Next, isolated cells undergo CRISPR editing in order to correct their genetic mutation or enhance the fetal hemoglobin. The modified cells were then infused back into the patients (this could also involve chemotherapy). These trials were designed to test the safety and success of the procedure. The findings of the trials were encouraging! They found that after the procedure, patients had significant increases in fetal hemoglobin levels, allowing for the symptoms of their condition, like SCD, to lessen. Additionally, some patients reported that they no longer required blood transfusions. The success of these trials are a huge milestone in the treatment for SCD and beta-thalassemia. Only mild negative reactions to the trials were noted, like a fever, and the effects of gene-editing are still being closely monitored to ensure maximum safety for patients.

To conclude, CRISPR technology has extensive potential to address various genetic disorders. As we saw, it creates new ways for treating conditions like SCD and beta-thalassemia. But, it also sets an example for future trials and research for other genetic disorders, like Huntington’s Disease, a neurodegenerative disorder caused by mutations in the HTT gene. The successful application we see in this example of the use of CRISPR may lead to further usage of gene-editing into medical practice. This form of treatment could replace previous ineffective solutions, and transform the well-being of patients with various diseases.

I’m passionate about this scientific breakthrough for sickle cell disease (SCD) and beta-thalassemia because of the amazing ways that CRISPR technology offers hope and shows promising results for symptom relief. I chose to write about this topic because I think it’s incredible that this form of gene editing could not only help patients managing SCD and beta-thalassemia, but thousands of patients dealing with a vast variety of diseases.

What are your thoughts on CRISPR and its potential impact on genetic diseases? Do you think we’ll see more advancements like this in the near future? I’d love to hear your opinions in the comments!

CRISPR-ing the Code: Deciphering Gene Regulation with Epigenome Editing

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On May 13, 2024

In Student Post

Let’s embark on a journey through the labyrinth of our genetic blueprint, scientists wield a powerful new tool, epigenome editing. Imagine having the ability to fine tune the orchestra of our genes, adjusting the volume of each instrument to compose the perfect symphony of life. In a groundbreaking study recently unveiled in Nature Genetics, researchers from the Hackett Group at EMBL Rome have unveiled a modular epigenome editing platform. This revolutionary system offers a glimpse into the intricate dance between our DNA and the proteins that regulate it, shedding light on how subtle molecular tweaks can orchestrate the grand narrative of biological existence. Join us as we delve into the captivating realm of chromatin modifications, CRISPR technology, and the tantalizing secrets they unveil about gene regulation.

CRISPR CAS9 technology

The researchers used CRISPR technology to precisely program nine important chromatin marks in the genome. The CRISPR technology served as the magic wand in the hands of researchers, enabling them to meticulously sculpt the epigenetic landscape of the genome. With CRISPR’s unparalleled precision and accuracy, scientists from the Hackett Group at EMBL Rome were able to program nine crucial chromatin marks at precise locations within the genome. This level of control allowed them to investigate the cause-and-consequence relationships between these chromatin modifications and gene regulation. CRISPR technology facilitates the development of reporter systems, which enable researchers to measure changes in gene expression at the single-cell level. This high-resolution analysis provides deeper insights into the dynamics of gene regulation and allows for the exploration of how different factors, such as chromatin structure and DNA sequence, interact to modulate gene activity. Additionally, CRISPR facilitated the creation of a ‘reporter system’, empowering researchers to measure changes in gene expression at the single-cell level. This enabled them to investigate the causal relationships between chromatin marks and gene regulation, shedding light on how these marks affect transcription, the process of copying genes into mRNA for protein synthesis. By employing a reporter system, they could measure changes in gene expression at the single-cell level and explore how DNA sequence influences the effects of each chromatin mark.

CRISPR Cas9 technology

Surprisingly, they discovered a new role for a chromatin mark called H3K4me3, which was previously thought to be a consequence of transcription. Their findings suggest a complex regulatory network involving multiple factors such as chromatin structure, DNA sequence, and genomic location.The researchers aim to further explore the implications of their findings by targeting genes across different cell types and at scale. This technology not only provides insights into the role of epigenetic changes in gene activity during development and disease but also offers potential applications in precision health by enabling the programming of desired gene expression levels.

The research conducted at he Hackett Group at EMBL Rome connects to a topic we have done in AP Biology. This is Gene Expression and Regulation. gene expression and regulation are fundamental concepts that delve into how genetic information stored in DNA is utilized by cells to produce proteins and carry out various functions. Here’s how the study conducted by scientists from the Hackett Group at EMBL Rome connects to gene expression and regulation in AP Biology. Chromatin Modifications and Transcription. The study investigates how chromatin modifications, such as histone methylation, influence the process of transcription, where genes are copied into mRNA molecules. This aligns with the AP Biology curriculum’s focus on understanding the role of chromatin structure in regulating access to DNA and controlling gene expression. Another way is Regulatory Mechanisms. The study provides insights into the regulatory mechanisms that govern gene expression by examining the causal relationships between chromatin marks and transcriptional activity. Students can learn about the intricate interplay between transcription factors, chromatin modifications, and regulatory DNA sequences in controlling gene expression levels.

Hope for Solving CRISPR Gene Editing’s Dangerous Side Effects: The ICP

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On May 3, 2024

In Student Post

While CRISPR,  “a technology that research scientists use to selectively modify the DNA of living organisms”, is an incredible advancement for humanity, it is far from perfect. In some treatments, the CRISPR treated cell or packaged CRISPR components are injected into patients with the goal of repairing diseased cells using precise gene edits. Gene therapies utilizing CRISPR have the potential to unintentionally induce “bystander” edits in various regions of the genome, occasionally resulting in the onset of new cancers or other diseases.  The intricacy of bodily tissue contains thousands of different cell types, which causes problems for scientists’ ability to correctly implement CRISPR technology.

CRISPR-Cas

Researchers from the University of California San Diego have developed new genetic systems to essentially fact check CRISPR gene edits. A sequence analyzer was made by this research team that is able to track on and off target mutations and the ways the genes are inherited from one generation to the next. Another system made called the Integrated Classifier Pipeline or ICP is able to reveal specific categories of mutations resulting from CRISPR editing. The ICP was developed in flies and mosquitoes, and it produces a “fingerprint” of how genetic materials are inherited. This allows scientists to track mutational edits to its source and risks associated with potentially dangerous CRISPR treatments.

The ICP and sequence analyzer could be the key to understanding how to further propel CRISPR technology and other “cutting-edge next-generation health technologies” to be consistently safe for human use.  According to Bier, a professor in the UC San Diego School of Biological Sciences, the CRISPR editing system can be more than 90% accurate, however, because it encodes ad nauseam its bound to have inconsistencies; The ICP is able to give a “high resolution picture” to describe what is going wrong.

In AP biology we learned about genetics. The bystander effect caused by CRIPSR  reminds me of gene mutations. The bystander effect is when surrounding genetic data of the gene or codons are deleted or rearranged. This is just like ‘normal’ gene mutations caused by errors in gene replication. Insertion, deletion, duplication, inversion, and translocation are all different gene mutations. The mutation’s effects can be silent, with no change to the amino acid; missense, altering one amino acid and potentially changing the protein’s function; or nonsense, causing premature termination of protein translation, resulting in a shorter or typically nonfunctional protein. The CRISPR bystander mutations would theoretically have the same effects as regular gene mutations.

The content that I have learned from AP Biology is setting me up to be able to understand modern complex biology related issues and discoveries that will continue to arise in the future. I wrote about this topic mainly for the bystander effect; I had never heard of the potential for negative side effects as a result of CRISPR treatment, so to learn and write about it was very interesting. Do you think CRISPR is safe to be used at this point? Will the ICP lead to a vast new bio-technology field of medicine? I think that CRISPR could be very useful to cancer patients who are terminally ill or cannot go through other forms of treatment.  The ICP also seems like a promising start to a new age of medicine and science. I  wonder if CRISPR technology could be used for treating plant disease, like the beech leaf disease, killing many trees in New York.

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