At CHOP, we are always looking for new and innovative ways to treat all neurological and muscular disorders. Currently, there are four of these such diseases that we can treat with experimental or FDA-approved targeted gene therapies.

• Duchenne muscular dystrophy (DMD)
• Spinal muscular atrophy (SMA)
• Cerebral adrenoleukodystrophy (CALD)
• Limb-girdle muscular dystrophy (LGMD)
• Metachromatic leukodystrophy (MLD)

The path from an idea in the lab to a clinical trial and finally to an FDA-approved therapy winds through these support programs. It’s with the expertise of these programs that discoveries are ushered through the complicated but critical steps toward safe and effective treatments.

• Clinical In Vivo Gene Therapy
• Cell and Gene Therapy Collaborative
• Cell Based Therapy Laboratory
• Cell and Gene Therapy Informatics
• Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT)
• Clinical Vector Core
• Research Vector Core
• Human Pluripotent Stem Cell Core
• Comparative Medicine Services Core
• Engineered Cell Therapy Core