Sickle Cell Disease gene therapy
A sickle- or crescent-shaped-shaped deficiency in the growth of red blood cells is the hallmark of sickle cell disease (SCD), a genetic disorder. Because of this flaw, the cells become clinging and firm, which obstructs tiny blood vessels and decreases the body's ability to carry oxygen. The result is often severe anemia, as the abnormal cells die prematurely, along with the potential for painful crises and serious complications. SCD is a continuous condition for individuals that suffer from it because it is inherited from one's parents. People with this crippling illness now have hope thanks to recent research breakthroughs, especially with the investigation of gene therapy as a possible treatment.
Changing the genetic composition of a patient's bone marrow cells to enable the production of normal red blood cells is the aim of gene therapy procedures. There are currently two exciting gene therapy procedures on the market that could result in an extended recovery period or even a successful cure for sickle cell disease. Patients who experience pain, swelling, and infections frequently may find that their quality of life is greatly improved by these therapies. Even though not every person with the gene experiences severe symptoms, these treatments are a huge advancement in the fight against sickle cell disease (SCD). They offer hope for a day when those who are affected by the condition may live longer, pain-free lives.
https://www.usnews.com/news/health-news/articles/2020-12-05/could-gene-therapy-cure-sickle-cell-disease-two-new-studies-raise-hopes
https://www.cdc.gov/ncbddd/sicklecell/facts.html
I remember discussing sickle cell disease in B&E last year. From what I remember, sickle cell disease might not only be painful for an individual but also could lead to other health complications such as strokes, organ damage, blood clots, infections, etc-- showing just how detrimental and problematic the disease is. So, if a potential treatment for SCD could be created in hopes of aiding in recovery from the disease, that is sensational!
ReplyDeleteI did look up some gene therapies for sickle cell disease, one being Casgevy and the other being Lyfgenia. Casgevy, an edit/ "cut," is made to a specific gene, which reactivates the production of fetal hemoglobin. This adulterates the "faulty" and or sickled red blood cells in the individual's bloodstream. Lyfgenia introduces a healthy hemoglobin-producing gene through a viral envelope. With all this being said, I am curious to see how this gene therapy, changing the genetic composition of a patient's bone marrow cells to produce normal-shaped red blood cells, compares to the other gene therapies currently used today to treat SCD. Furthermore, I am curious to see if this gene therapy will be as successful as what is being predicted.