Protease-Activatable Adeno-Associated Virus Vector for Gene Delivery to Damaged Heart Tissue.

ABSTRACT: Adeno-associated virus (AAV) has emerged as a promising gene delivery vector because of its non-pathogenicity, simple structure and genome, and low immunogenicity compared to other viruses. However, its adoption as a safe and effective delivery vector for certain diseases relies on altering its tropism to deliver transgenes to desired cell populations. To this end, we have developed a protease-activatable AAV vector, named provector, that responds to elevated extracellular protease activity commonly found in diseased tissue microenvironments. The AAV9-based provector is initially inactive, but then it can be switched on by matrix metalloproteinases (MMP)-2 and -9. Cryo-electron microscopy and image reconstruction reveal that the provector capsid is structurally similar to that of AAV9, wit

SUBMITTER: Guenther CM 

PROVIDER: S-EPMC6404099 | biostudies-literature | 2019 Mar

REPOSITORIES: biostudies-literature