Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome Editing

Title
Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome Editing
Authors
Keywords
genome editing, allele-specificity, TRIKAFTA, cystic fibrosis, CRISPR-Cas9, nonsense-mediated decay, CFTR
Journal
Molecular Therapy-Methods & Clinical Development
Volume 17, Issue -, Pages 1118-1128
Publisher
Elsevier BV
Online
2020-05-13
DOI
10.1016/j.omtm.2020.05.002

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