AAV Packaging Services
Gene therapy is a method of delivering therapeutic genes to cells or tissues using modified viruses or other technologies in order to address genetic disorders at their source. The understanding of how the adeno-associated virus (AAV) may be utilized as an efficient delivery mechanism for therapeutic genetic material into living tissue is one of the most exciting breakthroughs in contemporary medicine. AAV gene therapy has the potential to be used to treat a wide variety of illnesses.
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AAV is a virus that has not been shown to cause illness in humans.AAV cannot reproduce itself without external assistance, and so can not multiply in the body in the same way as conventional viruses do. This enables scientists to carefully regulate the amount of AAV administered.
AAV has surpassed all other forms of gene therapy in treating genetic disorders. AAV is at the forefront of therapeutic advancements today, having been employed in the development of the only two FDA-approved gene treatments now accessible.
To increase the efficiency and specificity of AAV infection of target tissues, researchers must genetically modify the viral capsid and generate mosaic vectors to create chimeric AAV by swapping domains or amino acids between serotypes. This may enable researchers to specifically target cells with certain serotypes in order to effectively transduce and express genes in a localized area.
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