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REMEDY: A Novel CRISPR-based Allele Specific Approach Corrects VCP Mutations

Author:D'Ambrosio, Eleonora   Ozes-Ak, Burcak   Sahenk, Zarife   Mendell, Jerry   Naeimi Kararoudi, Meisam   

Session Name:S21: Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S21.006  

Author Institution:Nationwide Children's Hospital, Columbus, OH