Therapeutic Gene Transfer for Blinding Diseases

Clinical trial success in a few gene therapy trials for inherited disorders, including one form of early onset retinal degeneration, has led to validation of the adeno-associated viral vector (AAV) platform. While AAV has several desirable properties and has proven safe and efficacious in these studies, several limitations of the vector system prevent its broad clinical application. 

It is exactly these limitations that are the focus of our studies which require a convergence  of virology, structural and molecular biology, engineering, clinical medicine and immunology. Specifically, we are interested in improving gene delivery of large genes, in working toward therapeutic gene editing approaches, to expand the cellular targets and safety of current vectors, and to develop simple molecular circuitry that allows exogenous induction and regulation of transgene expression for human gene therapy.

A second effort by members of the Vandenberghe lab is to build translational gene therapy programs for vision disorders targeting both inherited and complex disorders and both anterior and posterior segment.

Currently, our laboratory is moving forward with optogenetic therapies for retinal blinding disorders, as well as several programs in gene augmentation for single gene defects.

AAV Retinal Targeting following Subretinal Injection (Design: Peter Mallen)  

AAV Retinal Targeting following Subretinal Injection (Design: Peter Mallen)

 

Principal Investigator

Luk H. Vandenberghe, PhD

 

Funding

Luk H. Vandenberghe, PhD

Luk H. Vandenberghe, PhD