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Confirmatory Results

CRISPR-pass: Gene rescue of nonsense mutations using adenine base editors

Choongil Lee, Dong Hyun Jo, Gue-Ho Hwang, Jihyeon Yu, Jin Hyoung Kim, Se-eun Park, Jin-Soo Kim, Jeong Hun Kim, Sangsu Bae
doi: https://doi.org/10.1101/545723
Choongil Lee
1Department of Chemistry, Seoul National University, Seoul 08826, South Korea,
2Center for Genome Engineering, Institute for Basic Science, Seoul 08826, South Korea,
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Dong Hyun Jo
3Fight against Angiogenesis-Related Blindness (FARB) Laboratory, Clinical Research Institute, Seoul National University Hospital, Seoul 03080, South Korea,
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Gue-Ho Hwang
4Department of Chemistry, Hanyang University, Seoul 04763, South Korea,
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Jihyeon Yu
4Department of Chemistry, Hanyang University, Seoul 04763, South Korea,
5Research Institute for Convergence of Basic Sciences, Hanyang University, Seoul 04763, South Korea,
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Jin Hyoung Kim
3Fight against Angiogenesis-Related Blindness (FARB) Laboratory, Clinical Research Institute, Seoul National University Hospital, Seoul 03080, South Korea,
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Se-eun Park
4Department of Chemistry, Hanyang University, Seoul 04763, South Korea,
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Jin-Soo Kim
1Department of Chemistry, Seoul National University, Seoul 08826, South Korea,
2Center for Genome Engineering, Institute for Basic Science, Seoul 08826, South Korea,
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Jeong Hun Kim
3Fight against Angiogenesis-Related Blindness (FARB) Laboratory, Clinical Research Institute, Seoul National University Hospital, Seoul 03080, South Korea,
6Department of Biomedical Sciences, Seoul National University College of Medicine, Seoul 03080, South Korea,
7Department of Ophthalmology, Seoul National University College of Medicine, Seoul 03080, South Korea,
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  • For correspondence: steph25@snu.ac.kr sangsubae@hanyang.ac.kr
Sangsu Bae
4Department of Chemistry, Hanyang University, Seoul 04763, South Korea,
5Research Institute for Convergence of Basic Sciences, Hanyang University, Seoul 04763, South Korea,
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  • For correspondence: steph25@snu.ac.kr sangsubae@hanyang.ac.kr
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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.

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Posted February 10, 2019.
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CRISPR-pass: Gene rescue of nonsense mutations using adenine base editors
Choongil Lee, Dong Hyun Jo, Gue-Ho Hwang, Jihyeon Yu, Jin Hyoung Kim, Se-eun Park, Jin-Soo Kim, Jeong Hun Kim, Sangsu Bae
bioRxiv 545723; doi: https://doi.org/10.1101/545723
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CRISPR-pass: Gene rescue of nonsense mutations using adenine base editors
Choongil Lee, Dong Hyun Jo, Gue-Ho Hwang, Jihyeon Yu, Jin Hyoung Kim, Se-eun Park, Jin-Soo Kim, Jeong Hun Kim, Sangsu Bae
bioRxiv 545723; doi: https://doi.org/10.1101/545723

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