Gene Therapy Research and Projects
Areas of Focus
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Muscular dystrophies
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Neuromuscular diseases
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Peripheral neuropathies
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Lysosomal storage disorders
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Ischemia and perfusion injury
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Cancer and regenerative medicine
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AAV gene transfer vector development
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Muscle stem cells
Featured Research and Projects
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Phase I clinical trial for Duchenne muscular dystrophy (DMD). Recombinant AAV vector is being injected directly into the muscles of DMD patients in a double-blind randomized clinical trial, to establish maximum tolerated dose (see the Duchenne muscular dystrophy (DMD) clinical trial video ).
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Phase 1 Clinical Trial of rAAV1.tMCK.human-alpha-sarcoglycan Gene Vector in Limb Girdle Muscular Dystrophy type 2D (alpha-sarcoglycanopathy)
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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.
