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Paper Details

CRISPR-Cas9 homology-independent targeted integration of exons 1-19 restores full-length dystrophin in mice.
Mol Ther Methods Clin Dev
0
2023
-12 promoter, AAV genomes, CRISPR, Cas9, DNA fragment, Duchenne muscular dystrophy, MHCK7 promoter, SPc5, X-linked disorder, dystrophin, exon 2, exons, exons 1-19, full-length dystrophin, genomes, intron 19, mice, muscle, patients, pre-spliced mega-exon encoding, pre-spliced mega-exon encoding exons 1-19, target site, transcript
Author NameAffiliation
Gregory L WheelerThe Institute for Genomic Medicine, The Abigail Wexner Research Institute at Nationwide Children's Hospital
Benjamin J KellyThe Institute for Genomic Medicine, The Abigail Wexner Research Institute at Nationwide Children's Hospital
Benjamin J KellyThe Institute for Genomic Medicine, The Abigail Wexner Research Institute at Nationwide Children's Hospital
Peter WhiteThe Ohio State University
Peter WhiteThe Institute for Genomic Medicine, The Abigail Wexner Research Institute at Nationwide Children's Hospital
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Release 1.2