ArticleMolecular therapy : the journal of the American Society of Gene Therapy2019
CRISPR-Pass: Gene Rescue of Nonsense Mutations Using Adenine Base Editors.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
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Who cites it
31 citing papers in PubMed.
- Advances in CRISPR Base Editing: From Molecular Evolution to Therapeutic Applications in Genomic Medicine.Journal of cellular and molecular medicine · 2026Review
- Precision therapeutic tRNA rescue of nonsense mutation R166X inJournal of precision medicine (Amsterdam, Netherlands) · 2025Article
- Nonsense-mediated mRNA decay: a key regulatory system engaged in cancer.Cell communication and signaling : CCS · 2025Review
- Intravitreal adenine base editing of RS1 improves vision in a preclinical mouse model of retinoschisis.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Establishment and rescue of fibroblast cell lines carrying a nonsense mutation of RB1 by CRISPR-based base editing.Scientific reports · 2025Article
- Suppressor tRNA in gene therapy.Science China. Life sciences · 2024Review
- Base editing correction of OCRL in Lowe syndrome: ABE-mediated functional rescue in patient-derived fibroblasts.Human molecular genetics · 2024Article
- Therapeutic Nonsense Suppression Modalities: From Small Molecules to Nucleic Acid-Based Approaches.Biomedicines · 2024Review
- FunctionalScience advances · 2024Article
- Design and Engineering of Light-Induced Base Editors Facilitating Genome Editing with Enhanced Fidelity.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Article
- Modeling human cancer predisposition syndromes using CRISPR/Cas9 in human cell line models.Genes, chromosomes & cancer · 2023Review
- Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing.Molecular therapy. Nucleic acids · 2023Article
- Review
- Biotechnological Advances to Improve Abiotic Stress Tolerance in Crops.International journal of molecular sciences · 2022Review
- Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- State-of-the-Art in CRISPR Technology and Engineering Drought, Salinity, and Thermo-tolerant crop plants.Plant cell reports · 2022Review
- Review
- In vivo gene editing via homology-independent targeted integration for adrenoleukodystrophy treatment.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Precision genome editing using cytosine and adenine base editors in mammalian cells.Nature protocols · 2021Article
- Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing.Nature biomedical engineering · 2021Article
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Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
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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