Sickle Cell Disease - First Gene Editing Treatments!! (Casgevy and Lyfgenia) скачать в хорошем качестве

Sickle Cell Disease - First Gene Editing Treatments!! (Casgevy and Lyfgenia)

This is not medical advice. This is purely education at the best of our ability. Every person needs to have their own individualized care. As such, any concerns you have should be evaluated by your own healthcare provider and nothing from this video, as errors are possible. The reason these treatments are such a revolutionary approval is because prior treatments have been focused on using drugs similar to chemo or donor stem cell transplants. Both of those options have many adverse effects. Casgevy and Lyfgenia instead alter this path by extracting the patient's own stem cells and genetically modifying them to produce normal un-sickle cell shaped red blood cells. "The U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology (CRISPR-CAS9), signaling an innovative advancement in the field of gene therapy. Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S. It is most common in African Americans and, while less prevalent, also affects Hispanic Americans. The primary problem in sickle cell disease is a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body’s tissues. This mutation causes red blood cells to develop a crescent or “sickle” shape. These sickled red blood cells restrict the flow in blood vessels and limit oxygen delivery to the body’s tissues, leading to severe pain and organ damage called vaso-occlusive events (VOEs) or vaso-occlusive crises (VOCs). The recurrence of these events or crises can lead to life-threatening disabilities and/or early death." Sources: https://www.fda.gov/news-events/press... https://www.cdc.gov/ncbddd/sicklecell... https://www.uptodate.com/contents/exa... https://www.uptodate.com/contents/lov... https://www.hopkinsmedicine.org/healt.... https://my.clevelandclinic.org/health...