Chemical Epigenetic Regulation of Adeno-Associated Virus Delivered Transgenes.

ABSTRACT: Adeno-associated virus (AAV) is a powerful gene therapy vector that has been used in several FDA-approved therapies as well as in multiple clinical trials. This vector has high therapeutic versatility with the ability to deliver genetic payloads to a variety of human tissue types, yet there is currently a lack of transgene expression control once the virus is administered. There are also times when transgene expression is too low for the desired therapeutic outcome, necessitating high viral dose administration resulting in possible immunological complications. Herein, we validate a chemically controllable AAV transgene expression technology in vitro that utilizes bifunctional molecules known as chemical epigenetic modifiers (CEMs). These compounds employ endogenous epigenetic machin

SUBMITTER: Umana JD 

PROVIDER: S-EPMC10517330 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature