CRISPR-Cas9 homology-independent targeted integration of exons 1-19 restores full-length dystrophin in mice.

Autor: Stephenson AA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Nicolau S; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Vetter TA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA., Dufresne GP; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Frair EC; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Sarff JE; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Wheeler GL; The Institute for Genomic Medicine, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Kelly BJ; The Institute for Genomic Medicine, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., White P; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA.; The Institute for Genomic Medicine, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Flanigan KM; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA.; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Aug 18; Vol. 30, pp. 486-499. Date of Electronic Publication: 2023 Aug 18 (Print Publication: 2023).