"Self-Regulating Aav Vectors For Safe Expression Of Mecp2 In Rett Syndrome" in Patent Application Approval Process (USPTO 20230416779).

A patent application has been filed by inventors from the University of Massachusetts for a self-regulating adeno-associated virus (AAV) vector that could potentially treat Rett syndrome, a neurological disease caused by mutations in the MECP2 gene. The inventors have discovered that certain combinations of miRNA regulatory elements can control the expression of a transgene within a specific range, which could help prevent off-target transgene toxicity. The patent application describes methods of engineering a transgene and recombinant nucleic acids with miRNA binding sites, as well as viral vectors and compositions for the treatment of Rett syndrome. The application provides detailed descriptions of these methods and their potential applications in gene therapy. [Extracted from the article]