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Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

Adeno-Associated Virus (AAV)

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Adeno-Associated Virus (AAV)

A member of the parvovirus family, which is composed of small viruses with a genome of a single-stranded DNA. AAVs insert genomic material at a specific site on human chromosome 19 with nearly 100% certainty, and are used to construct vectors that introduce genes into cultured cells. There is virtually no downside to AAV vectors, given the small amount of DNA they can carry and because AAV is non-pathogenic (most people are AAV carriers). In contrast to adenoviruses, AAV usually does not trigger an immune response to cells infected with it, and thus can deliver genes to sites of interest, including the brain in the context of gene therapy for diseases of muscle and eye, tissues where AAV seems to be most useful.
Segen's Medical Dictionary. © 2012 Farlex, Inc. All rights reserved.

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