Constructing and evaluating caspase-activatable adeno-associated virus vector for gene delivery to the injured heart.

ABSTRACT: Adeno-associated virus (AAV) is a promising vector for gene therapy, but its broad tropism can be detrimental if the transgene being delivered is harmful when expressed ubiquitously in the body, i.e. in non-target tissues. Delivering the transgene of interest to target cells at levels high enough to be therapeutically effective while maintaining safety by minimizing delivery to off-target cells is a prevalent challenge in the field of gene therapy. We have developed a protease activatable vector (provector) platform based on AAV9 that can be injected systemically to deliver therapeutic transgenes site-specifically to diseased cells by responding to extracellular proteases present at the disease site. The provector platform consists of a peptide insertion into the virus capsid which disrupt

SUBMITTER: Brun MJ 

PROVIDER: S-EPMC7770761 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature