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Paper Details

Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells.
Mol Ther Nucleic Acids
1
2023
5' GT and 3' AG nucleotides, A, CFTR, Canonical splice, Cas9, EMG, Protospacer, adenine, c, c.2988+, c.2988+1G, canonical splice site variant, cystic fibrosis, cystic fibrosis transmembrane conductance regulator (), cystic fibrosis transmembrane conductance regulator () gene, expression minigene, human, human airway epithelial cells, introns, mRNA, protospacer adjacent motifs, protospacers, single-guide RNAs
Author NameAffiliation
Gregory A NewbyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard
Gregory A NewbyHoward Hughes Medical Institute, Harvard University
Gregory A NewbyHarvard University
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard
David R LiuHoward Hughes Medical Institute, Harvard University
David R LiuHarvard University
Garry R CuttingJohns Hopkins University School of Medicine Baltimore
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