The Gene Therapy Program encompasses research aimed at developing basic and applied gene therapy for genetic metabolic diseases. Specific areas of support include:
- Studies to improve gene delivery systems;
- Studies of the basic biology of AAV, adenovirus, retrovirus, and lentivirus vectors;
- Studies of nonviral methods of gene transfer such as liposomes or DNA conjugates;
- Studies to target gene delivery to specific cell types;
- Studies of gene therapy of stem cells to treat a genetic metabolic disease.
For further information, contact Dr. Catherine McKeon, Senior Advisor for Genetic Research.