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Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]
The concept of gene therapy is to fix a genetic problem at its source. If, for instance, a mutation in a certain gene causes the production of a dysfunctional protein resulting (usually recessively) in an inherited disease, gene therapy could be used to deliver a copy of this gene that does not contain the deleterious mutation and thereby ...
Substrate reduction therapy is FDA approved and there is at least one treatment available on the market. [10] Gene therapy aims to replace a missing protein in the body through the use of vectors, usually viral vectors. [11] In gene therapy, a gene encoding for a certain protein is inserted into a vector. [11]
The following is a list of genetic disorders and if known, type of mutation and for the chromosome involved. Although the parlance "disease-causing gene" is common, it is the occurrence of an abnormality in the parents that causes the impairment to develop within the child. There are over 6,000 known genetic disorders in humans.
(Reuters) -AstraZeneca said on Friday its Alexion unit had agreed to buy U.S. drugmaker Pfizer's early-stage rare disease gene therapy portfolio for up to $1 billion, plus royalties on sales, as ...
Pages in category "Approved gene therapies" The following 25 pages are in this category, out of 25 total. This list may not reflect recent changes. A.
Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) has announced longer-term data from the Glycogen Storage Disease Type Ia (GSDIa) and Ornithine Transcarbamylase (OTC) Deficiency Phase 1/2 studies.
Genetic studies indicate that all domestic cats descend from the Middle Eastern wildcat, Felis sylvestris lybica. ... Some of the benefits of cat therapy include help with depression via ...
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