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This treatment thus avoids the need for regular, costly blood transfusions. [13] [14] In December 2023, the FDA approved the first gene therapy in the US to treat patients with Sickle Cell Disease (SCD). The FDA approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of SCD. [57]
Sickle-cell anemia, which can cause blood cells to clump up and block blood vessels, can also lead to stroke. Stroke is the second leading cause of death in people under 20 with sickle-cell anemia. [52] Air pollution may also increase stroke risk. [53]
This is an accepted version of this page This is the latest accepted revision, reviewed on 19 September 2024. Science of genes, heredity, and variation in living organisms This article is about the general scientific term. For the scientific journal, see Genetics (journal). For a more accessible and less technical introduction to this topic, see Introduction to genetics. For the Meghan Trainor ...
Management of multiple sclerosis. Multiple sclerosis (MS) is a chronic inflammatory demyelinating disease that affects the central nervous system (CNS). Several therapies for it exist, although there is no known cure. The most common initial course of the disease is the relapsing-remitting subtype, which is characterized by unpredictable ...
Socioeconomic factors are the related cause, alongside it presenting barriers to treatment in the disease. [30] Sickle cell disease is more susceptible to be found in those of descent from places such as those in the Mediterranean, Italy, Turkey, and Greece, as well as Africa and regions of South and Central America. [48]
A typical human cell contains about 150,000 bases that have suffered oxidative damage. [85] Of these oxidative lesions, the most dangerous are double-strand breaks, as these are difficult to repair and can produce point mutations , insertions , deletions from the DNA sequence, and chromosomal translocations . [ 86 ]
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