Abstract
Gene therapy using adeno-associated viral (AAV) vectors has shown early promise in the clinic, particularly for retinal gene therapy. The therapeutic transgene cassette can be packaged into different AAV capsid variants, each having a unique transduction profile.
At present, AAV capsid serotype selection for a specific clinical trial is based on effectiveness in small animal models. Substantial progress has been made in improving effectiveness of gene therapy for eye diseases in rodents. This includes selection of the vector and promoter combination and testing phenotypic outcomes in relevant model systems.
Here, I will describe recent work from my group on developing gene therapy and gene delivery strategies for treatment of retinitis pigmentosa. Our innovative therapies aim to restore vision in late stages of the disease with a special focus on high acuity vision.
