[EZ-Cytox] CRISPR-Pass: Gene Rescue of Nonsense Mutations Using Adenine Base Editors
- - 짧은주소 : http://dogenbio.com/bbs/?t=96
- - 년도 : 2019
- - 제품명 : EZ-Cytox
- - 학술지명 : Molecular Therapy
- - 주소링크 : https://www.sciencedirect.com/science/article/pii/S1525001619302278
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.