Gene Therapy Research and Projects

Areas of Focus

• Muscular dystrophies

• Neuromuscular diseases

• Peripheral neuropathies

• Lysosomal storage disorders

• Ischemia and perfusion injury

• Cancer and regenerative medicine

• AAV gene transfer vector development

• Muscle stem cells

Featured Research and Projects

• Phase I clinical trials of a rAAV based HIV-1 vaccine in collaboration with Children’s Hospital of Philadelphia, the International AIDS Vaccine Initiative, NIH (NIAID) and Targeted Genetics Corp.

• Translational research of novel treatment strategies for muscular dystrophino-pathies using genes that correct or modify the disease phenotype (e.g. micro-dystrophin, galgt2, follistatin, exon-skip inducing RNA and RNAi).

• Clinical and pre-clinical development of novel, neurotrophic-based treatment strategies for amyotrophic lateral sclerosis (ALS). Viral vector mediated gene transfer is being utilized to provide factors for neuronal growth and protection of affected motor neurons.

• Research into second-generation gene transfer rAAV vectors targeting multiple organs, that allow for delivery through the vasculature.

• Research in the isolation, growth and engraftment potential of muscle derived stem cells to treat a host of degenerative muscle diseases.

• Pre-clinical studies for the treatment of the lysosomal storage disorder MPS IIIB using viral mediated gene transfer systemically or targeted to the central nervous system (CNS).

• cGMP Viral Vector Manufacturing Facility to support Phase I and Phase II clinical trials.