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Two species of AAV were recognised by the International Committee on Taxonomy of Viruses in 2013: adeno-associated dependoparvovirus A (formerly AAV-1, −2, −3 and −4) and adeno-associated dependoparvovirus B (formerly AAV-5). [66] Until the 1990s, virtually all AAV biology was studied using AAV serotype 2.
Self-complementary adeno-associated virus (scAAV) is a viral vector engineered from the naturally occurring adeno-associated virus (AAV) to be used as a tool for gene therapy. [1] Use of recombinant AAV (rAAV) has been successful in clinical trials addressing a variety of diseases. [ 2 ]
Recombinant adeno-associated virus (rAAV) based genome engineering is a genome editing platform centered on the use of recombinant AAV vectors that enables insertion, deletion or substitution of DNA sequences into the genomes of live mammalian cells.
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Kenneth Ira Berns (June 14, 1938 – January 26, 2024) was an American virologist who was a distinguished professor emeritus at the department of Molecular Genetics and Microbiology at the University of Florida College of Medicine. [1]
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A new gene is inserted into a cell using the AAV protein shell. Once inside the nucleus, the new gene makes functional protein to treat a disease. Alipogene tiparvovec , sold under the brand name Glybera , is a gene therapy treatment designed to reverse lipoprotein lipase deficiency (LPLD), a rare recessive disorder, due to mutations in LPL ...