BioQuakes

AP Biology class blog for discussing current research in Biology

Tag: treatment

CRISPR Technology leads the way for potential breakthrough in cancer treatment

According to The American Cancer Society, scientists can alter the structure of a particular white blood cell known as the T-cell.  This method, known as CAR T-cell therapy, has long been established as a potential weapon against cancer, altering T-cells to best fight cancer based on the patient’s own characteristics.  According to an article in Forbes, the genetic editing procedure that has been used to facilitate this technology has relied upon “Viral Vectors,” which according to Beckman Coulter, viral vectors are modified viruses “that can be used to deliver nucleic acids into the genetic makeup of cells.”  While useful, Forbes asserts that the usage of Viral Vectors can be time-consuming and “can cost up to $50,000 per dose.”  For these reasons, scientists have looked towards a new technology, known as CRISPR technology to facilitate the editing of T-cells.CRISPR logo

 

According to the National Human Genome Research Institute, “CRISPR (short for “clustered regulatory interspaced short palindromic repeats”) is a technology that research scientists use to selectively modify the DNA of living organisms.”  According to Forbes, this technology differs from viral vector technology in that it involves the synthesis of “RNA guides,” which allow the scientists to break a DNA sequence at a targeted point, allowing for a change, as would be required to facilitate CAR T-cell therapy.  Furthermore, the article asserts that “synthesizing an RNA guide is cheaper and more efficient than cultivating retroviral vectors,” potentially allowing for the treatment to be more widespread.  As stated in the Forbes article by William A. Haseltine, former professor at Harvard University, “there is potential to propel CAR T design forward by integrating contemporary innovations such as CRISPR/Cas9 technology.”  It is therefore clear that the usage of CRISPR technology for CAR T-cell therapy could revolutionize cancer treatment

 

 

Many of the concepts referenced in this post involve concepts we have learned in AP bio class.  For example, in the immune system section of the cell communication unit, we learned about the various types of T-cells.  For example, we learned how T-killer cells kill infected cells, such as cancer cells, T-memory cells retain information to prevent further infection, and T-helper cells stimulate other T-cells.  From here, we learned how T-cells, more specifically T-killer cells, can be used to fight cancer, which connects to CAR T-cell therapy’s usage of the cells for gene editing. 

 

While CRISPR technology’s use in CAR T-cell therapy is exciting, according to Haseltine, it “still has room for improvement.”  This technology is not fully developed, and will probably need years to be widespread.  But still, the complete implementation of CRISPR technology in CAR T-cell therapy remains an exciting prospect.

Self-Assembling Hydrophobic Sandwiches

You read that correctly! Researchers at Rice University in Houston, Texas alongside Jeffrey Hartgerink have made a significant advance in injury treatment, illness education, and drug candidate by testing the self-assembling abilities of 3D printed nanofibrous multidomain peptide hydrogels, referred to as “hydrophobic sandwiches.” 

Hydrogel

The main goal of Hartgerink’s team was to create a structure that could house cells and help them grow tissue by 3D printing the peptide ink. The printing allows researchers to recreate the complexity of biological structures due to their soft and flexible tissue-like feel, making this a major scientifical discovery and advantage. Hartgerink and his team describe their printed peptides as “hydrophobic sandwiches” due to their design, flexibility, and behavior. The peptides were printed to have one hydrophobic side and one hydrophilic side, allowing them to flip on top of each other when placed in water and resemble sandwiches. Like we learned in AP Biology, the hydrophillic qualities of one side will attract water, and the hydrophobic qualities of the other will repel water. Hydrophobic molecules repel water because they are nonpolar molecules, so they are not attracted to water, which is polar. Once the “sandwiches” were stacked after flipping in the water, they formed the hydrogels which can be vital to tissue engineering and wastewater treatments. 

Hydrogel Structure

The multidomain peptides have already been utilized due to their self-assembling nature for regenerating nerves, treating cancer, healing wounds, and encouraging tissue development throughout the body. Rather than only focusing on this aspect of the peptides, Adam Farsheed, a lead author in Hartgerink’s study, wanted to specifically highlight the fact that these peptides are an ideal 3D-printing ink choice due to their self-assembling nature. When testing the “sandwiches,” Farsheed took a unique, brute-force approach to add more of the material, rather than chemically modifying it, to test its function and ability to reassemble itself after deformation. He proved that adding more peptide material lets the peptide reassemble and heal itself extremely well after being deformed. This discovery will make the hydrogels an ideal candidate for scientific and medical usage.  

Through continued testing, he was also able to confirm that the peptides behave differently depending on their charge. The peptide cells with a negative charge tended to ball up on the substrate of the experiment and the positively charged cells spread out and started to mature on their own. Farsheed has confidently stated that their findings will allow the group to “control cell behavior using both structural and chemical complexity.” Both Hartgerink and Farsheed have made incredible contributions to the world of science through their studies using 3D-printed peptide hydrogels. 

 

CRISPR Mini | New Territory Unlocked

For over a million years, DNA has centered itself as the building block of life. On one hand, DNA (and the genes DNA makes up) shapes organisms with regard to physical appearance or ways one perceives the world through such senses as vision. However, DNA may also prove problematic, causing sickness/disease either through inherited traits or mutations. For many years, scientists have focused on remedies that indirectly target these harmful mutations. For example, a mutation that causes cancer may be treated through chemotherapy or radiation, where both good and bad cells are killed to stop unchecked cell replication. However, a new area of research, CRISPR, approaches such problems with a new perspective.

The treatment CRISPR arose to answer the question: what if scientists could edit DNA? This technology involves two key components – a guide RNA and a CAS9 protein. Scientists design a guide RNA that locates a specific target area on a strand of DNA. This guide RNA is attached to a CAS9 protein, a molecular scissor that removes the desired DNA nucleotides upon locating them. Thus, this method unlocks the door to edit and replace sequences in DNA and, subsequently, the ways such coding physically manifests itself. Moreover, researchers at Stanford University believe they have further broadened CRISPR’s horizon with their discovery of a way to engineer a smaller and more accessible CRISPR technology.

This study aimed to fix one of CRISPR’s major flaws – it is too large to function in smaller cells, tissues, and organisms. Specifically, the focus of the study was finding a smaller Cas protein that was still effective in mammalian cells. The CRISPR system generally uses a Cas9 protein, which is made of 1000-1500 amino acids. However, researchers experimented with a Cas12f protein which contained only 400-700 amino acids. Here, the new CasMINI only had 529 amino acids. Still, the researchers needed to figure out if this simple protein, which had only existed in Archaea, could be effective in mammals that had more complicated DNA.

To determine whether Cas12f could function in mammals, researchers located mutations in the protein that seemed promising for CRISPR. The goal was for a variant to activate a protein in a cell, turning it green, as this signaled a working variant. After heavy bioengineering, almost all the cells turned green under a microscope. Thus, put together with a guide RNA, CasMINI has been found to work in lab experiments with editing human cells. Indeed, the system was effective throughout the vast majority of tests. While there are still pushes to shrink the mini CRISPR further through a focus on creating a smaller guide RNA, this new technology has already opened the door to a variety of opportunities. I am hopeful that this new system will better the general well-being as a widespread cure to sickness and disease. Though CRISPR, and especially its mini version, are new tools in need of much experimentation, their early findings hint at a future where humans can pave a new path forward in science.

What do you think? Does this small CRISPR technology unlock a new realm of possibility or does it merely shed light on scientists’ lack of control over the world around us?

The Promise of Messenger RNA Therapy

A recent article about messenger RNA therapy outlines the evolution of messenger RNA therapy and how it has gone from an idea to a globally used treatment in just the past seventeen years. Recently, messenger RNA therapies such as the Pfizer-BioNTech and Moderna COVID-19 vaccines have been used by hundreds of millions of people around the world. 

The author Drew Weissman, a vaccine research professor at the University of Pennsylvania, and his colleague Katalin Karikó created mRNA molecules back in 2005 that would not cause harm when injected into animal tissue. Then in 2017, Weissman and Norbert Pardi found that this mRNA creation could be brought into human cells through a fatlike nanoparticle without harm, and that bringing this modified mRNA in protect mRNA from being broken down by the body and resulted in the immune system generating antibodies and more effectively neutralize the invading virus. Vaccines-09-00065-g001This mRNA fatlike nanoparticle is known as mRNA-LNP (pictured to the left). mRNA is able to enter cells without harm because it is carried in by this liquid nanoparticle which is known for its role in transportation. The Pfizer-BioNTech and Moderna COVID-19 vaccines use this mRNA-LNP, and in clinical trials have shown to successfully prevent over 90% of treated people from contracting COVID-19.

The positive results from many trials and studies of the Pfizer-BioNTech and Moderna COVID-19 vaccines have provided a lot of information on the success of mRNA-LNP. It has been found that mRNA-LNP is much more effective and quicker than other approaches to COVID-19 treatments such as growing vaccines in laboratory cell cultures. 

41541 2020 159 Fig1 HTMLMessenger RNA therapy works by making cells create proteins that induce a reaction from the immune system in response to invading viruses (pictured to the right). This reaction in response to invading viruses is called the humoral response, where cytotoxic T cells are made to release proteins that destroy infected cells. The humoral response also trains the immune system to respond to and attack that virus in the future by creating memory B cells to recognize it, which is called a secondary immune response.  This method of instructing cells to create these proteins yields a greater quantity at a time that conventional protein and monoclonal antibody therapies. 

The success of messenger RNA therapy in COVID-19 vaccines has inspired the further research and use of this method for other viruses, as well as cancers, food, allergies, and autoimmune diseases, and many clinical trials are underway. Messenger RNA therapy could be a much more time and cost efficient alternative for a lot of conditions and treatments. More research still needs to be done, and there are many improvements that could be made (such as smaller doses of or a better supply chain for the vaccine), but overall messenger RNA therapy is very promising for treatments of the future.

Cellular GPS: A New Cancer Treatment

In recent years, it is estimated that 40% of people will face cancer during their lifetime. Still, there exist few reliable treatments for cancer, whereby it has become one of the leading causes of death in the world. Ideally, if a tumor is confined to one area of the body and is easily accessible, doctors may simply try to remove it with surgery. However, tumors are usually widespread and not so easily identifiable, whereby doctors turn to treatments such as chemotherapy which causes mass death of both healthy and unhealthy cells throughout the body. Nonetheless, scientists have discovered a potentially more targeted treatment for cancer, involving guiding magnetic seeds to tumors and burning them.

Bodily cells undergo the cell cycle, a controlled series of stages referred to as interphase, mitosis, and cytokinesis. Interphase is comprised of the G1, S, and G2 phases where cells perform normal activities, grow, undergo DNA replication, and duplicate organelles. Next, mitosis marks the division of the nucleus while cytokinesis marks the division of the cytoplasm. During this process, there are “checkpoints” at the end of the G1 phase, G2 phase, and mitosis. For example, maturation-promoting factors may trigger a cell’s passage through the G2 checkpoint if it has successfully duplicated and grown or stop a cell’s passage through this gateway if it has incorrectly copied itself. Cancer is caused when mutations in certain genes cause uncontrollable cell growth; this unchecked and rapid division causes many cells to pack closely together into tumors which hijack bodily functions, ultimately proving fatal unless treated.

Recently, researchers have proposed a new method to treat cancer patients, especially those with tumors in hard-to-reach places like the cranium. This treatment would send a highly magnetic thermoseed into one’s body which would be remotely heated once at the site of the tumor. Here, like driving a car on a loopy road, a doctor would use an MRI scanner to carefully guide the magnetic seed through the patient’s body. MRI scanners are reliable tools in scanning the location of tumors, so they would accurately pinpoint where to target and where to avoid with the thermoseed. Thus, this controlled method of eradicating tumors poses less of a threat with regard to damaging the body as a whole or even damaging surrounding tissues.

Although the prospect of such innovative research for remedies fuels optimism, it surely raises the question of which patients should undergo the new thermoseed treatment rather than well-trusted treatments like chemotherapy or open surgery. According to the study, this method would be greatly influential in treating glioblastoma, a common brain cancer. With traditional open brain surgeries, patients merely survive a year to a year and a half on average. Moreover, side effects are always a large risk with many current cancer treatments. However, I believe that killing the tumor remotely with a thermoseed and MRI has the potential to be a breakthrough, successfully eliminating the tumor and posing fewer long-lasting effects. While this treatment is still an idea at the beginning stages of research, its projected benefits make me optimistic about its future.

What do you think? Will this proposed cancer treatment be the reliable cure scientists have been looking for or a futile treatment that only reminds us of the challenge we are up against?

Can HCQ(Hydroxychloroquine) Prevent COVID-19 Infection and Help Recovery? The Research Says “No”

Have you ever wondered what chemicals and such are being used to treat and illness you have? HydroxychloroquineWell, for treatment of the COVID-19 vaccine, one of the chemicals used is hydroxychloroquine (HCQ), also known as Plaquenil. HCQ is a immunosuppressive drug and anti-parasite that can treat and prevent malaria, lupus, and arthritis.

HCQ was used as pre-exposure prophylaxis against COVID-19 infection in healthcare workers as a study. There were 1294 participants from ages 24-38 with 61% being women. 273 (21.1%) of the participants were healthcare workers but still 83 (6.4%) of them tested positive after duty. This showed that the use of HCQ had no effect on the prevention of the COVID-19 virus.

What made hydroxychloroquine an option used in preventing COVID-19 in the first place? Symptoms of coronavirus disease 2019 2.0There are typically four phases of a more severe version of COVID-19. The first phase would be the incubation period that has a median of 5.1 days. After that is the second phase which lasts around 5-10 days where flu-like symptoms arise. These include, fever, cough, muscle pain/soreness, fatigue, nausea, and diarrhea. Up until the second phase, the severity of the illness can be considered normal. After the second phase, there is normally a progression to a hyperinflammatory acute respiratory distress syndrome (ARDS). ARDS is a life-threatening lung injury that makes breathing difficult. As the second phase progresses onto the third phase, ARDS causes dyspnea, tachypnea, and sometimes hypoxemia making a person extremely out of breath and in need for hospital care. During this third phase, a person affected severely of COVID-19 will normally have high fevers with elevated inflammatory markers and progressive formation of organ failure. For some of these severe cases of COVID-19, effective treatments were desperately needed.

From data of previous epidemics, HCQ have been widely used around the world for Ebola, H7N9 influenza, and SARS virus infection. HCQ has been used to treat a number of auto-immune diseases by raising intracellular pH and affect endosomal activity. However, in the case of COVID-19, HCQ has no positive effect in preventing the coronavirus and may even cause more harm to our bodies.

Fimmu-11-01648-g004

As an immunosuppressive drug, it made sense to give HCQ to patients with early onset of COVID-19 and as a pre-exposure prophylaxis. HCQ impacts cytokine production and suppresses antigen presentation. The medication was used in various ways: as an oral medication by itself to take before contracting COVID-19, taking it after contracting COVID-19, and combined therapy with azithromycin. None of these ways had a surprising result in preventing COVID-19 or with helping a person recover. It was then believed that the impact of cytokine production and suppression of antigen presentation may cause immunologic consequences resulting in the hampering of the innate and adaptive antiviral immune response, possible making it more dangerous with patients with COVID-19. It has been determined that HCQ is not suitable for the treatment of COVID-19.

The process of proving HCQ effectiveness in fighting COVID-19 can be related to AP Biology because of the way the medication works with the immune system. Hydroxychloroquine is a medication that can raise intracellular pH and affect endosomal activity. Acidity of cell pH and endosomes are topics that we learned first quarter. In relation to the second quarter, HCQ is a immunosuppressive drug known to impact cytokine production and antigen presentation. Cytokines and antigens are part of the innate and adaptive immune system that we learned about recently.

The COVID-19 Treatment Pill: Destroying or Amplifying The Virus?

After seeing millions of people die from COVID-19, a new discovery has been found that could be the first long term treatment option to give patients suffering from COVID-19 a chance to fight it off, but how sure can we be that the treatment pill will work?

Pill 1

New data about an antiviral pill made by Merck with its partner Ridgeback Pharmaceuticals show that the treatment pill is not as stellar as first believed. The drug has drawbacks that could outweigh its potential to fight the coronavirus and keep people out of the hospital.

The U.S. Food and Drug Administration is now deciding whether to grant emergency use authorization for the drug called molnupiravir, after the agency’s advisory panel narrowly voted to recommend it on November 30. The drug was authorized to be of use in the United Kingdom on November 4, and if the FDA follows suit, it could wind up being just a temporary treatment. Some advisers have already urged the agency to be ready to withdraw the authorization as soon as something better comes along.

Finding an early treatment for COVID-19 hasn’t been easy due to the constant trial and error that scientists keep facing, so when the development of molnupiravir came out a lot of experts hailed it as they thought it could be a potential game changer for the pandemic. It would be utilized as a pill that could be given to people early in the infection, keep health care systems from being overwhelmed, and spare people at high risk from the most severe complications. 

In a clinical trial, the drug showed early signs of preventing hospitalization and death from COVID-19 in people who are at high risk. In fact, the results were so promising — a 48 percent reduction in the relative risk of hospitalization or death — that the trial was stopped so that the drug might potentially reach the public earlier.

But on November 26, Merck announced in a news release that when all the available data from the trial was in, the reduction in relative risk fell to 30 percent against hospitalization and death compared with a placebo. The shift stemmed from an unexplained decrease in severe disease among people in the placebo group in the last part of the trial.

Overall, among the 709 people in the molnupiravir group, there were 48 hospitalizations and one death compared with 68 hospitalizations and nine deaths among the 699 people who got a placebo, dropping the effectiveness from the initial 48 percent to 30 percent.

Taking that lower-than-expected efficacy into account, the FDA’s antimicrobial drugs advisory committee came to a split 13–10 decision about whether the antiviral drug should be granted emergency use authorization, with experts on each side of the vote often agreeing with points made by the opposing side. The debate and vote reflected a storm of uncertainty about the drug’s efficacy and who should use it — the list of people who would not be eligible is far longer than those most experts would give the drug to. The panel also looked into whether the drug could lead to even more dangerous versions of the coronavirus, whether it can cause growth delays in children or mutations in human DNA, and other unanswered questions.

The antiviral pill works by making mutations in viral RNA so that viruses are rendered noninfectious and eventually stop replicating. Such mutations happen throughout the virus’s genetic instruction book, or genome.

SARS-CoV-2 without background

Some of those mutations could land in the spike protein, which helps the coronavirus break into cells, or other proteins and make the virus more transmissible or more evasive to vaccines. As learned in AP Biology, the spike proteins enable the host cell to be taken over by the virus and multiply and infect the surrounding cells. The vaccine that is being administered to people all around the world contains the antibodies that you would get if you were to be infected with the COVID-19 virus, so that if you were to get the virus, your body would go into its secondary immune response. This is when the memory cells facilitate a faster, stronger and longer response to the same COVID-19 antigen. Getting the vaccine would protect your body from having a life threatening reaction to the virus. If there is a mutation that lands on the spike protein, then the vaccine will be of no use to people since that is a completely different makeup of the virus. That’s especially a fear if people don’t finish the full five-day course of the drug needed to render the virus inoperable, leading potentially to highly mutated new forms of the virus that could infect others. 

Merck representatives said that possibility is unlikely, because after five days of taking even a half dose of the drug, infectious viruses were no longer detectable among study participants tested. In one study, the company found seven patients who had changes in the coronavirus’s spike protein after taking molnupiravir, but there was no evidence that the viruses spread to other people or affected the patient’s health.

Molnupiravir might also create mutations in human DNA, researchers say. The drug is a nucleoside analog — an artificial RNA building block that can mimic the bases cytosine and uracil. Some enzymes in human cells might convert those RNA subunits to a DNA building block, which may lead to mutations in human DNA, especially in rapidly reproducing cells, such as blood cells. How likely that is is an open question.

Still, there are no good remedies for people with mild to moderate COVID-19. As of November 30, more than 82,000 people in the United States are being diagnosed with COVID-19 each day and more than 800 die. Those numbers are expected to increase as case counts surge in some parts of the country. The new omicron variant might add fuel to that fire if it proves more contagious than the currently dominant delta variant.

So even with all of molnupiravir’s drawbacks, federal regulators might decide a 30 percent reduction in hospitalizations and deaths is worth giving the drug temporary authorization.

The drug might be helpful for “the right patient population, the right virus at the right time,” said Lindsey Baden, an infectious diseases doctor at Brigham and Women’s Hospital in Boston who chaired the FDA’s advisory committee. “To me that at least suggests there are populations where there may be benefit.”

But more studies need to be done to address concerns about the drug, he said. “It’s the absence of data that makes many of us uncomfortable.”

President Joe Biden said December 2 during remarks laying out a plan to combat the omicron variant that the government has secured a supply of the drugs and, if authorized, will distribute them similarly to vaccines.

 

The Potential End To COVID-19: How An Antiviral Pill Could Decrease Death Rates

When will the world return back to normal? In recent years, people have questioned the longevity of the COVID-19 outbreak. While concentrating on vaccine delivery and vaccination capabilities, a pill has been developed in the hopes of preventing future COVID variations. Hopefully, the pill will eventually be administered to patients; this would make it the first oral treatment for the virus.

A current study on molnupiravir, an antiviral pill, has published data demonstrating that the medicine has the ability to lower hospitalization and fatality rates as a result of COVID-19. The study dealt with two groups of people. One group of 377 people were given a placebo, and the other group of 385 people were given molnupiravir to examine how the antiviral affected patients with COVID-19. The findings were substantial. Within 29 days of starting the trial, 14.1 percent of the group given the placebo were hospitalized. Fortunately, of the individuals who were given molnupiravir, only 7.3 percent of them were hospitalized.

Molnupiravir is a prodrug of N4-hydroxycytidine (NHC), a nucleoside analog (meaning that it contains a sugar and a nitrogenous base). Molnupiravir metabolism

Molnupiravir is similar to the genetic coding of the coronavirus’s RNA, as is remdesivir (a FDA-approved medication). By interfering with the polymerase enzyme, the “fake” basic elements impair the coronavirus’s RNA synthesis, preventing the virus from replicating. Despite the fact that the two medications serve the same goal, they serve different actions. Remdesivir penetrates a growing RNA strand, slowing and ultimately blocking the polymerase enzyme. Unlike the COVID-19 vaccine, the structure of molnupiravir gives it the ability to target the polymerase enzyme instead of the virus’s spike protein. Molnupiravir enters the cell and is transformed into RNA-like building components. The active medication binds to the genome of RNA viruses, setting off a chain of mutations; this process is known as viral error catastrophe. In simpler terms, it disrupts how the virus replicates RNA.

Molnupiravir could theoretically be administered as soon as a patient receives a positive COVID-19 test, thereby preventing floods of COVID-19 patients from overburdening medical systems while the highly infectious delta variant continues to spread. Although the side effects of the drug remain unknown, it has been reported that the side effects of COVID-19 are much worse than those of molnupiravir. The antiviral drug has the potential to save lives, but the primary concern is about the long-term repercussions. When contemplating molnupiravir, the fear of birth abnormalities or cancer comes into play because it is a mutagenic medication. In response, the drug’s creator, Merck, stated that there is no indication of the possibility for mutagenicity. Although the manufacturer is confident in the treatment and believes that the long-term consequences are insignificant, it is logical that parents might have concerns about molnupiravir.

Ultimately, if patients receive the vaccination that targets the spike protein and are also able to take molnupiravir, hospitalization and mortality rates may dramatically reduce.

I Have a Gut Feeling that Microbiomes Are the Next Step in Medicine

We as humans are a very genetically diverse species. But what if we could find microorganisms that have over 150 times more genes right within our own guts. Scientists believe that human microbiomes is the key to treating diseases in the future and some analysts believe that the field of human microbiome market will reach $3.2 billion by 2024.

Lack of microbiome diversity has shown to cause diseases like MS, diabetes, and asthma. So microbiomes have demonstrated themselves to be a key component of our health. To better understand these links, many projects launched in the past few years have focused on mapping microbial genesthat are associated with disease. For example, there are certain microbiomes that make cancer drugs ineffective whereas others are actually necessary to make these drugs work. Thus, a patient’s microbiome makeup directly correlates to their survival chances. So the next step in the Microbiome market is modifying the microbiome to work for the patient.

One older technique of doing this is traced back to China thousands of years ago where they would transplant whole microbial communities to treat diarrhea also known as Fecal microbiota transplant (FMT). A more defined approach is where beneficial bacterial strains are delivered, alive, to the patient’s gut.

In the UK, a company called Microbiotica transfers non-pathogenic strains of C. difficile to fight C. difficileinfections, IBD, and cancer. Even though these approaches have drawn a lot of attention to the field, some scientists argue that the bacteria we ingest are not as well adapted to living in our gut as those that have been living there for years thus are not effective.

In France, an opposite approach is used by Enterome where drugs target specific bacteria leaving the rest to the gut microbiome intact. This approach is aimed at treating Chron’s diseaseand cancer.

Another approach is using bacteriophages to kill specific bacteria strains. Eligo Bioscience takes it a step further by using the bacteriophages to deliver CRISPR/Cas9 into the bacteria to kill it by cutting the DNA of the strain carrying the disease only.

Finally, certain companies like Blue Turtle Bio engineer bacteria to make them produce drugs directly within the human gut.

Since this is a rather new field, there are newly emerging companies along with groups who do not believe in the practice. Personally, I do believe in this practice because there is already a strong correlation between our immune system and specific microbiome strands. With microbiome treatments comes microbiome diagnostics to determine which patients can benefit from these therapies. Thus a high number of approaches will initially fail to treat disease. So far, gut infections and inflammatory diseases seem to be strongly correlated to the gut microbiota, so that will be an area where more research should be applied.  A crucial challenge for the field will be to move from correlation to causation, and a lot of research is still needed for that.

 

Preferential Gene Expression: Not As Random As We Thought

Our conventional knowledge of genetics dictates that the activation of genes in our DNA is random. It is equally likely that our body will express our mother’s alleles as it is that our body will express our father’s. In the case that one parent donates a defective copy, it will be silenced; the other parent’s healthy set of DNA takes precedence and becomes activated.

However, a new study indicates that gene expression and activation is not as random as we thought. In certain regions of the body, our genes demonstrate preferential expression.

A team of scientists at the University of Utah found that almost 85 percent of genes in juvenile mice brains displayed preferential treatment. The mice brains activated one parent’s set of DNA over the other’s. This phenomenon was observed in other areas of the body, as well as in primates.

Although the preferential expression came to a close within ten days, it could provide explanations for vulnerability to brain diseases such as schizophrenia, ADD, and Huntington’s. The temporary preferential treatment to one parent’s copy of DNA could trigger a host of problems specific to that cell site that lead to such disorders, if the parent had given a defective copy of genes.

The study has the potential to alter our basic understandings of genetics, and how we are more prone to certain specialized diseases.

Image: (Public Domain, https://pixabay.com/en/dna-biology-medicine-gene-163466/)

CRISPR-Cas9 Providing New Treatment Possibilities

The genetic editing tool, CRISPR-Cas9, is making greater strides regarding RNA linked diseases. The knowledge of how CRISPR-Cas9 can affect DNA has increased over the past couple of years. By targeting the DNA with CRISPR-Cas9 scientists have found new ways to modify protein production and treat certain diseases, which led to editing genes. However, now there is inquiry about what would occur if CRISPR-Cas9 targeted RNA.  Many diseases are linked to RNA and by targeting RNA with CRISPR-Cas9 we could find new treatments to fight off cancer, autism, and X-syndrome. Researchers at University of California, San Diego School of Medicine have been able to accomplish targeting the RNA. Gene Yeo, PhD, associate professor of cellular and molecular medicine hopes to use this technique to fix RNA behavioral diseases.

PDB_1wj9_EBI

Image Source

RNA can affect when and where proteins will be produced, but if the RNA transport is deficient than it can cause diseases from autism to cancer. Evaluating RNA movement will allow new treatments to be found.  Yeo and colleagues at the University of California, Berkeley, have created RCas9, which is targeting RNA in live cells. They were able to do so by altering certain features of the CRISPR-Cas9. A short nucleic acid, PAMmer, that they designed was used to direct CRISPR-Cas9 to an RNA molecule. They then targeted RNA that encodes certain proteins which were ACTB, TFRC, and CCNA2. The CRISPR-Cas9 would combine with a fluorescent protein to reveal the movement of RNA into stress granules. This allowed the team to track RNA through the live cells without using artificial tags, which are normally used to track RNA.

CRISPR-Cas9 is opening new ways to find out more information to fix diseases regarding DNA and now RNA. There has been controversy regarding CRISPR-Cas9 because it is a tool to edit genetic material, but in this case it is helping us fight off diseases that have been affecting lives for ages. Do you believe that CRISPR-Cas9 should only be used for certain cases or that people should be able to use it freely?

Original Article

Other sources:

1.https://health.ucsd.edu/news/releases/Pages/2016-03-17-CRISPR-Cas9-targets-RNA-in-live-cells.aspx

2. http://www.techtimes.com/articles/142061/20160318/gene-editing-tool-crispr-cas9-can-now-monitor-and-target-rna-in-living-cells.htm

 

 

Editing the Brain Using Epigenetic Tools

Epigenetics is a huge part of our life and influences us in ways we may not be aware of. Did you know that it is impossible to create and save new memories without epigenetic tags? The brain is heavily reliant on Epigenetics to do its functions, and this makes it a huge topic of research to figure out the ways in which the epigenetics of the brain could affect certain diseases or memory. Recently special epigenetic molecular tools have been created that can erase specific epigenetic markers throughout the genome. The possible effects these tools could have on the curing of diseases of the brain or psychological ailments are tremendous.

These “epigenetic editing” procedure use either CRISPR (clustered, regularly interspaced, short, palindromic repeats) or TALE (Transcription activator-like effector) systems of modification. These systems can carry an Epigenome modifying enzyme and deliver it a specific site they are programmed to go to. This allows researchers to target very specific epigenetic changes and either shut them down or turn them on and possibly determine their correlation with certain ailments of the brain. “We’re going from simply being able to observe changes to being able to manipulate and recapitulate those changes in a controlled way,” Day said. This quote from Day, one of the researchers of this project, shows that we advance from only being able to observe epigenetic influences on the brain, to being able to manipulate and control them to potential aid us in combating diseases.

Researchers can catalog all of the epigenetic changes involved in forming and preserving a new memory. If we are able to track these epigenetic changes, then could we implant memories in to a person’s mind, by copying similar epigenetic changes? These researchers where also able to trigger not only the place where epigenetic change happens, but also the exact time using optogenetics. This form of using light to control neurons allows researchers to use the TALE system and a light switch apply epigenetic change to very specific brain regions or cell types.

One of the final goals of this research is to eventually be able to use epigenetic as a form of therapy to benefit PTSD, depression, schizophrenia, and cognitive function using the ability to alter epigenetic marks. This can also be used in a similar way to silence mutated genes that are damaging the cells or the body as whole. This form of using TALE and CRISPR to alter epigenetic tags creates a lot of hope for PTSD, depression, schizophrenia, Alzheimer’s, Parkinson’s, Huntington’s and other similar disease treatment options.

New Breast Cancer Gene Discovered

 

 

 

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Today, one of the most talked about cancers is breast cancer. Breast cancer is defined as cancer that forms in the tissues of the breast. There are two types of breast cancer: ductal carcinoma, which is most common and begins in the lining of the milk ducts (thin tubes that carry milk from the lobules of the breast to the nipple) and lobular carcinoma, which begins in the lobules (milk glands) of the breast.

According to a new study done by the Wellcome Trust Sanger Institute and University of Cambridge, a gene has been identified to have a major association in aggressive subtypes of breast cancer. The research suggests that an overactive BCL11A gene causes the development of tripe-negative breast cancer.

The study was conducted in human cells and in mice. The study was important because one in five patients are affected by triple-negative breast cancer. From the conducted research, Dr. Walid Khaled discovered that by adding an active human BCL11A gene to a human or a mouse’s breast cells (in the lab) caused them to behave as cancer cells. Increasingly, Dr. Khaled concluded that “by increasing BCL11A activity we increase cancer-like behaviour; by reducing it, we reduce cancer-like behavior.”

This research and study is extremely important because from the results, the team was able to propose that BCL11A is a strong candidate for development of a possible targeted treatment. Typical treatments of breast cancer include radiation and chemotherapy as well as surgery. The most known surgeries are Lumpectomy/partial mastectomy (large portion of the breast is removed) and a full mastectomy (full removal of breasts)

I chose this article because I know many dear friends that have faced and survived the battle of breast cancer. I believe that spreading awareness and screening early is extremely important. In addition, I am very hopeful that new advances will be made so that others need not endure the excruciating fight of breast cancer.

 

The Rise of NFL Brain Injuries and Possible Solutions

tackling leading to brain injuries

tackling leading to brain injuries

Brain-injury treatment program that was created for military veterans hurt on the battlefield has now been updated to include professional athletes. Eisenhower Center’s representatives announced that it will be the main facility used by the NFL Players Association to treat brain and neurological issues. This will be facilitated through the After the Impact program.

Residential neuro-rehab facility is located in Ann Arbor. The program has intense treatment for athletes recovering from concussions, mild traumatic brain injury, or PTSD. It came from Eisenhower Center’s transitional treatment program to serve military soldiers dealing with brain injuries.

The NFL expects 28% of retired players to suffer Alzheimer’s or moderate dementia; this has caused former players to sue. About a third of all retired NFL players will suffer long-term cognitive problems and the After the Impact program has helped some reach the path to recovery.

NFL provoked a judge to approve a $1 billion settlement of concussion lawsuits ignoring concerns raised by former players. The 65-year fund will resolve thousands of lawsuits on the NFL, accusing them of hiding their knowledge of concussions and brain injuries in an attempt to keep players.

I picked this topic because I watch football every weekend and have wondered about the impact of the hitting on the players. It is nice to see that there have been efforts made to fix this.

Sources: http://www.huffingtonpost.com/2014/12/16/braininjury-program-worki_n_6337018.html?utm_hp_ref=brain

http://espn.go.com/nfl/story/_/id/11905362/nfl-urges-judge-approve-estimated-1-billion-settlement-concussion-lawsuits

http://www.eisenhowercenter.com/neurobehavioral

 

Big News for Retina Pigmentosa Patients

 

Photo by luisar

In a recent article, it was revealed that patients with the rare disease retinitis pigmentosa, may be able to find treatment using the Argus II Retinal Prosthesis System. Retinitis Pigmentosa is a rare, only about 100,000 Americans have it, inherited retinal disease that “causes the breakdown of cells in the retina.”

Dr. Robert Greenberg, CEO of Second Sight (the company that created the Argus II Retinal Prosthesis System), stated that those with retinal pigmentosa are “looking down a tunnel that gradually narrows until it disappears entirely.”

The Argus II uses a “video camera and a transmitter mounted on a pair of glasses” to help the individual. “Images are then wirelessly transmitted to electrodes implanted into the patient’s retina.” While the Argus II will not restore vision completely, it will allow users to partially regain vision. Tests show that the Argus II helped patients perform daily activities with ease. These activities included “navigating sidewalks and curbs, matching different color socks, and recognizing large words in sentences.”

Personally, I think this is a great technological leap. This prosthesis system will hopefully reach out to those with retinal pigmentosa and others with macular degeneration.

Trial for New ALS Treatment Failed

Photo by: Nemo

Biogen Idec, a drug developing company, has recently discontinued their work on a new drug that was, hopefully, going to help patients with Amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig’s disease. A recent article explained that a new drug, known as dexpramipexole, was not effective in the phase 3 trial of the study.

Amyotrophic lateral sclerosis (ALS) is a disease where nerve cells “waste away and die.” These cells are unable to send messages to muscles, therefore symptoms include paralysis and muscle weakness. The progression of the disease is slow and “once the patient loses the function of muscles in the chest area, it becomes hard to breathe.” There is no known cure for this disease but scientists are looking for ways to prolong the disease.

Biogen Idec believed that the drug, dexpramipexole, was hopefully going to “slow the progression of loss of muscle function and prolong the lives of people with the disease.” While the phase 3 trial was not successful, the phase 2 trial of patients receiving dexpramipexole showed some success. 50% of the patients, in the second trial, showed a slower decline of muscular function. This was a big accomplishment for Biogen Idec but the phase 3 was not as effective. Therefore, Biogen Idec’s study involving a new treatment for ALS ended.

Even though Biogen Idec’s study was not effective, other companies have successfully found a way to slow the progression of ALS. Thus far, only one drug has been approved to help patients with ALS. This drug is known as Rilutek/Riluzole and it is only modestly effective.

Doctors are in need of a new drug that will help patients with ALS. I think its great that companies like Biogen Idec are involved in finding a way to treat this rare disease. I hope that researchers will use the information from the failed trial to find another way to treat ALS.

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