Patent Issued for Modified AAV constructs and uses thereof (USPTO 12054715).

A patent has been issued to the University of Massachusetts for modified adeno-associated virus (AAV) constructs and their uses in gene therapy. The patent describes methods and compositions that increase the replication and packaging efficiency of AAV vectors containing hairpin-forming RNA cassettes, which are valuable tools for gene function studies and gene therapy applications. The invention is based on the discovery that DNA fragments encoding RNA hairpin structures can serve a function similar to a mutant inverted terminal repeat (ITR) during viral genome replication, generating self-complementary vector genomes. This technology allows for efficient, safe, and sustained in vivo gene silencing. [Extracted from the article]