Areas of Focus
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.