Base editing as a genetic treatment for spinal muscular atrophy.

Alves, Christiano R R; Ha, Leillani L; Yaworski, Rebecca; Lazzarotto, Cicera R; Christie, Kathleen A; Reilly, Aoife; Beauvais, Ariane; Doll, Roman M; de la Cruz, Demitri; Maguire, Casey A; Swoboda, Kathryn J; Tsai, Shengdar Q; Kothary, Rashmi; Kleinstiver, Benjamin P.

Afiliación

Alves CRR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.
Ha LL; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.
Yaworski R; Department of Neurology, Harvard Medical School, Boston, MA, USA.
Lazzarotto CR; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.
Christie KA; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.
Reilly A; Ottawa Hospital Research Institute, Ottawa, ON, Canada.
Beauvais A; Department of Cellular and Molecular Medicine, University of Ottawa, ON, Canada.
Doll RM; Centre for Neuromuscular Disease, University of Ottawa, ON, Canada.
de la Cruz D; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Maguire CA; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.
Swoboda KJ; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.
Tsai SQ; Department of Pathology, Harvard Medical School, Boston, MA, USA.
Kothary R; Ottawa Hospital Research Institute, Ottawa, ON, Canada.
Kleinstiver BP; Department of Cellular and Molecular Medicine, University of Ottawa, ON, Canada.