Two gene therapies were recently approved by the Food and Drug Administration (FDA) to treat sickle cell disease: Casegevy from CRISPR/Vertex and Lyfgenia from bluebird bio. You probably have questions about these new treatment options. Read more below. Is gene therapy a cure for sickle cell disease? Gene therapy is a potentially curative therapy. This means that it could act as a cure, but it is too new to say for sure. It causes a big decline in pain episodes, but we need to learn more about long-term impacts and side effects. It is also not a “one-and-done” treatment. The FDA currently recommends 15 years of patient follow up.
Sickle Cell Disease
The most common kind of an inherited blood problem, sickle cell disease, affects around 70,000 to 100,000 Americans. This condition, which affects affected persons from birth, results in the creation of aberrant hemoglobin. The hemoglobin molecule, which is found inside red blood cells, normally binds to oxygen in the lungs and transports it to all regions of the body. Healthy red blood cells are flexible enough to pass through even the smallest blood vessels. The hemoglobin in sickle cell disease is defective, causing the red blood cells to be inflexible and formed like a "C" or sickle, from which the condition gets its name. Sickle cells can become entrapped and impede blood flow, resulting in discomfort and infection. Sickle cell disease complications emerge when sickled cells impede blood flow to specific organs. Stroke, acute chest syndrome (a disorder that reduces the level of oxygen in the blood), organ damage, other disabilities, and, in some circumstances, premature death are among the most serious effects.
To inherit sickle cell disease, both parents must have sickle cell disease (two sickle cell genes) or sickle cell trait (one sickle cell gene). There are varieties of sickle cell disease known as sickle C or sickle thalassemia, which are both significant illnesses but can be less severe at times. If you have sickle cell disease, you will carry one sickle cell gene to your children.
Executive Directors note
Sickle Cell Disease has always been a part of our family, and will always be a beloved cause I will be a strong advocate for. In 2013 a family member transitioned from complications due to Sickle Cell Disease. Since then, I’ve gone on to honor her memory by becoming a SCD Community Health Worker, participating in a host of activities, workshops, conferences, classes nationwide, and founded the only statewide Sickle Cell community based organization Sickle Cell Warriors of Wisconsin- SCWWI. As a respected pillar of the community, Entrepreneur, and business owner I plan to continue to use my voice, and platform to bring awareness, and education to community members, the medical community, and allies who are able to support our mission and vision. I would like for you to consider partnering with us as we journey ahead to break the sickle cycle.
Non-Profit Organization Director
SCWWI is a 501(c)3 organization dedicated to advocating,educating, providing support while exercising compassion and creating mentorship opportunities for individuals living with Sickle Cell Disease.
Sickle Cell Warriors of Wisconsin Blood Drive
A Book Signing Event
James Griffin book signing, connecting the drops community event hosted by Versiti Blood Center of Wisconsin
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