![]() It may be possible to use gene correction in the treatment of a wide range of diseases recent experimental work has used gene editing technologies to extract HIV from the genome of affected laboratory mice and to excise the expanded region responsible for Huntington’s disease from the human gene. The goal of gene correction is to produce a protein that functions in a normal manner instead of in a way that contributes to disease. CRISPR/cas9, TALEN or ZFN) to remove repeated or faulty elements of a gene, or to replace a damaged or dysfunctional region of DNA. ![]() Gene correction can be achieved by modifying part of a gene using recently-developed gene editing technology (e.g. Therapies based on gene addition are being developed to treat many diseases, including adenosine deaminase severe combined immunodeficiency (ADA- SCID), congenital blindness, hemophilia, Leber’s congenital amaurosis, lysosomal storage diseases, X-linked chronic granulomatous disease, and many others. Most often, a modified virus such as adeno-associated virus (AAV) is used to carry the gene into the cells. Gene addition involves inserting a new copy of a gene into the target cells to produce more of a protein. ![]() Currently, these techniques are mainly used to target specific populations of somatic cells. The five main therapeutic strategies are presented below. Additionally, almost any gene in the human genome can be targeted, so the potential for new therapies is immense. Yes! At the time of writing, lists more than 1000 different types of gene therapy in clinical trials.
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