BioQuakes

AP Biology class blog for discussing current research in Biology

Modifying Genes to Cure a Blood Disease?

Helen Bolando, a 16 year old living with sickle cell disease, recently became the youngest recipient of an experimental treatment at Boston Children’s Hospital. This treatment made her the youngest person to have her DNA manipulated in hopes of reversing sickle cell’s effects. 

What is sickle cell disease?

Sickle cell disease is a disorder caused by a gene mutation that causes the shape of blood cells to resemble that of a crescent. Characteristics of sickle disease include a low red blood cell count and frequent infections. Due to their shape, blood cells in individuals with sickle cell cells break down too early, causing a lack thereof. This lack of blood cells is known as sickle cell anemia and causes a multitude of symptoms ranging from fatigue and shortness of breath, to delayed growth in children. Painful episodes are also common due to the shape of the red blood cells. Their crescent shape causes blockages in blood vessels, depriving organs and tissues of oxygen, sometimes leading to organ failure. 

A new gene therapy?

Researchers at Boston Children’s Hospital have found that hemoglobin genes (genes found in the blood) are only active in the preceding red blood cells. These genes are only active for 4-5 days before red blood cells mature and when they’re active, they communicate with other cells through communication such as long distance signaling, as we’ve learned earlier in our bio class . The question for researchers is as follows: “How do you manipulate a gene, or put a gene in, so it is expressed only in those cells and at high levels?”  New treatments to solve this burning question include the extraction of immature blood cells from patient’s bone marrow. These stem cells are then genetically modified and re-infused in hopes of creating new, healthy blood cells. Even more interestingly, scientists have found that fetal blood cells have an absence of sickle cells and are testing ways to block the gene that stops fetal hemoglobin production and begins that of adult hemoglobin.Bluebird Bio, a biotech company in Cambridge, Mass conducted a study during which nine patients were treated with gene therapy. Results stated that four patients of the nine who were  treated at least six months earlier, produced enough hemoglobin to no longer have the symptoms of sickle-cell disease!

Researchers are making incredible strides in solving this painful disease using extremely creative and innovative techniques! Are there any other methods of solving sickle cell disease you can think of  based on what we’ve learned so far about cell communication? 

 

 

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4 Comments

  1. metalibolism

    This is so interesting. Another positive for this gene therapy is that the more common treatment requires a matching donor to replace the stem cells in the bone marrow according to https://sicklecellanemianews.com/sickle-cell-disease-treatments/ while gene therapy does not require a donor. Stem cell transplantation is not available to all people because of the difficulty in finding a donor, but successful gene therapy should allow the patient to produce their own healthy blood cells.

  2. nukellyicacid

    Great article! I’m glad that there is a potential treatment for sickle cell anemia using gene therapy. The current procedures such as stem cell treatment is a risky process, while others are not relatively effective. Furthermore, it’s very interesting how we can connect what we learned about cell signaling to research that is created in the present day. I’ve attached an article that discusses how a man in Alabama became completely free of sickle cell disease due to receiving gene therapy from a clinical trial. https://www.webmd.com/a-to-z-guides/news/20190917/alabama-man-free-of-sickle-cell-after-gene-therapy

  3. angtigen

    I enjoyed your article kylsquared. I didn’t realize efforts were being made to reverse the effects of sickle cells. Upon doing research I learned there is an experimental therapy being done which is a specialized bone marrow transplant. High doses of chemotherapy are used to destroy all of a child’s bone marrow, and then replaced with a healthy bone marrow from a donor. Transplant recipients often require harsh immunosuppressants for years. Research is still being done to improve this transplant procedure. Do you think there are better techniques to curing this disease that aren’t as invasive?
    https://newsinhealth.nih.gov/2014/08/transplant-reverses-sickle-cell-disease

  4. angtigen

    I enjoyed your article Kyla. I didn’t realize efforts were being made to reverse the effects of sickle cells. Upon doing research I learned there is an experimental therapy being done which is a specialized bone marrow transplant. High doses of chemotherapy are used to destroy all of a child’s bone marrow, and then replaced with a healthy bone marrow from a donor. Transplant recipients often require harsh immunosuppressants for years. Research is still being done to improve this transplant procedure. Do you think there are better techniques to curing this disease that aren’t as invasive?
    https://newsinhealth.nih.gov/2014/08/transplant-reverses-sickle-cell-disease

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