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Safety and Efficacy of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping

Author:Shieh, Perry   Campbell, Craig   Deconink, Nicolas   De Waele, Liesbeth   Flanigan, Kevin   Lorentzos, Michelle   Phan, Han   Mix, Chris   Ray, Soma   Wang, Dazhe   Dugar, Ashish   Kerr, Douglas   Naylor, Maria   Guglieri, Michela   

Session Name:S31: Hot Topics in Child Neurology  

Topic:Child Neurology and Developmental Neurology  

Program Number:S31.010  

Author Institution:University of California Los Angeles, Los Angeles, CA  London Health Sciences Centre, London, ON, Canada  Neuromuscular Reference Center UZ Gent, Gent, Belgium  University Hospitals Leuven, Leuven, Belgium  Nationwide CHildrens Hospital, Columbus, OH  Children's Hospital at Westmead, Westmead, New South Wales, Australia  Rare Disease Research, LLC, Atlanta, GA  Dyne Therapeutics, Inc., Waltham, MA  Royal Victoria Infirmary, Newcastle University, Newcastle-Upon-Tyne, United Kingdom  

Correction of Duchenne Muscular Dystrophy Mutations by CRISPR/Cas9 Gene Editing Using Homology-Independent Targeted Integration

Author:Nicolau, Stefan   Vetter, Tatyana   Stephenson, Anthony   Dufresne, Gabrielle   Frair, Emma   Trecha, Breanna   Sarff, Jessica   Flanigan, Kevin   

Session Name:P7: Child Neurology and Developmental Neurology: Neuromuscular 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.006  

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