Recombinant adeno-associated viral vectors are the leading platform for treating human diseases through gene delivery.
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Updated on July 10th, 2023
Gene therapies are groundbreaking approaches utilizing viral and non-viral vectors. These vectors introduce therapeutic genes into patient cells to treat diseases, especially genetic disorders. In clinical trials, adeno-associated viral (AAV) vector-based treatments have emerged as the preferred platform in viral vector gene therapies due to their unique safety and efficacy features.
AAV Vectors
AAVs are single-stranded DNA viruses that require co-infection with other viruses, mainly adenoviruses, to replicate. When used as gene therapy vehicles, researchers generate recombinant AAVs (rAAVs) that contain the DNA for the gene of interest and lack viral genes, packaged into capsids.1 To form these infectious particles in the laboratory, genes necessary for AAV replication and formation are supplied in trans so that they are ...
