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

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

Nonsense-mediated decay (NMD) is a major pathogenic mechanism underlying a diversity of genetic disorders. Nonsense variants tend to lead to more severe disease phenotypes and are often difficult targets for small molecule therapeutic development as a result of insufficient protein production. The t...

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Bibliographic Details
Main Authors: Steven Erwood, Onofrio Laselva, Teija M.I. Bily, Reid A. Brewer, Alexandra H. Rutherford, Christine E. Bear, Evgueni A. Ivakine
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
genome editing
allele-specificity
TRIKAFTA
cystic fibrosis
CRISPR-Cas9
nonsense-mediated decay
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300899

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