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Rare Disease Patient Advocacy Perspectives on the Promise and Challenges of Gene Therapy

Kristin Voorhees, Kim McCleary, Kenneth Hobby,Marcus Landgren,Iris Ferrecchia, Kirsty Hoyle, Juana I. Navarrete, Amanda Jaksha, Kristin Smith,Rupal N. Gupta,Eric Crombez

MOLECULAR THERAPY（2023）

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