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R and Molecular Therapeutics at The Children s Hospital of Philadelphia is testing two cutting-edge gene transfer approaches to treatment in Phase 1 clinical trials. Focused on hemophilia and a form of blindness, the trials are both evaluating the safety and tolerability of the approaches. One of only a few such programs based at pediatric institutions, the center targets new gene, stem cell and tissue-engineering findings that have the greatest potential impact to translate into medical innov.

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