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CRISPR-Pass: Gene Rescue of Nonsense Mutations Using Adenine Base Editors
Cited 31 time in
Web of Science
Cited 32 time in Scopus
- Authors
- Issue Date
- 2019-08
- Publisher
- Nature Publishing Group
- Citation
- Molecular Therapy, Vol.27 No.8, pp.1364-1371
- Abstract
- A nonsense mutation is a substitutive mutation in a DNA sequence that causes a premature termination during translation and produces stalled proteins, resulting in dysfunction of a gene. Although it usually induces severe genetic disorders, there are no definite methods for inducing read through of premature termination codons (PTCs). Here, we present a targeted tool for bypassing PTCs, named CRISPR-pass, that uses CRISPR-mediated adenine base editors. CRISPR-pass, which should be applicable to 95.5% of clinically significant nonsense mutations in the ClinVar database, rescues protein synthesis in patient-derived fibroblasts, suggesting potential clinical utility.
- ISSN
- 1525-0016
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