PT - JOURNAL ARTICLE AU - Choongil Lee AU - Dong Hyun Jo AU - Gue-Ho Hwang AU - Jihyeon Yu AU - Jin Hyoung Kim AU - Se-eun Park AU - Jin-Soo Kim AU - Jeong Hun Kim AU - Sangsu Bae TI - CRISPR-pass: Gene rescue of nonsense mutations using adenine base editors AID - 10.1101/545723 DP - 2019 Jan 01 TA - bioRxiv PG - 545723 4099 - http://biorxiv.org/content/early/2019/02/10/545723.short 4100 - http://biorxiv.org/content/early/2019/02/10/545723.full AB - 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.