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Wednesday, September 1, 2021

08-31-2021-2219 - Adeno-associated viruses (AAV) virus primate Dependoparvovirus Parvoviridae 20nm 20 replication-defective, nonenveloped viruses kilobase vector gene therapy agent isogenic human disease model retina adeno immune

 

Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus Dependoparvovirus, which in turn belongs to the family Parvoviridae. They are small (20 nmreplication-defectivenonenveloped viruses and have linear single-stranded DNA(ssDNA) genome of approximately 4.8 kilobases (kb).[1][2]

AAV are not currently known to cause disease. The viruses cause a very mild immune response. Several additional features make AAV an attractive candidate for creating viral vectors for gene therapy, and for the creation of isogenic human disease models.[3] Gene therapy vectors using AAV can infect both dividing and quiescent cells and persist in an extrachromosomal state without integrating into the genome of the host cell, although in the native virus integration of virally carried genes into the host genome does occur.[4] Integration can be important for certain applications, but can also have unwanted consequences. Recent human clinical trials using AAV for gene therapy in the retina have shown promise.[5]

https://en.wikipedia.org/wiki/Adeno-associated_virus

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