AAV-ancing gene therapies

The most common vector used in FDA-approved gene therapies is the adeno-associated virus (AAV). They are widely favored due to their safety profile, ability to target specific tissues and long-term gene expression. This article explores the biology of AAV vectors, their applications in gene therapy, challenges, and future directions. What are AAV vectors? Adeno-associated viruses are small, non-enveloped viruses belonging to the Parvoviridae family. They are naturally replication-deficient, requiring a helper virus (such as adenovirus or herpesvirus) to replicate, making AAV non-pathogenic in humans, a key factor in its safety profile. Structurally, AAV vectors consist of two main components: the capsid...