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Regulatory Pathway for Gene Therapy for Rare Diseases: An Illustrative Example from the PaVe-GT Platform Program

Rodica Stan, Richa Madan Lomash, Randy J. Chandler, Oleg Shchelochkov, Charles P. Venditti, Carsten G. Bonnemann, Philip J. Brooks, Elizabeth Ottinger

MOLECULAR THERAPY（2023）

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