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Multi-Center Blinded Preclinical Efficacy Study Shows Significant Differences in Promoter Performance for Gene Replacement Therapy of SMARD1/CMT2S

Julieth A. Sierra Delgado,Shibi Likhite,Vicki McGovern,Amy Huffenberger,Deepti Chugh,Amy N. Hicks,Sarah E. Holbrook,Monica Nizzardo,Cassandra Dennys-Rivers,Shrestha Sinha Ray, Stefania Corti,Arthur Burghes,W. David Arnold,Gregory A. Cox,Kathrin Meyer

MOLECULAR THERAPY（2020）

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