ArticleMolecular therapy. Nucleic acids2017
Targeted Disruption of V600E-Mutant BRAF Gene by CRISPR-Cpf1.
Article in Molecular therapy. Nucleic acids, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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Who cites it
23 citing papers in PubMed.
- CRISPR/Cas9 in Cancer Therapy: Precision Genome Editing Approaches Targeting Hematological Malignancies and Solid Tumors Through Cellular, Biochemical, and Molecular Mechanisms.Cell biochemistry and biophysics · 2026Review
- CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges.Molecular therapy. Nucleic acids · 2026Review
- Trends and challenges of AAV-delivered gene editing therapeutics for CNS disorders: Implications for neurodegenerative disease.Molecular therapy. Nucleic acids · 2025Review
- BRAFiScience · 2025Article
- BRAFiScience · 2025Article
- Robust genome editing activity and the applications of enhanced miniature CRISPR-Cas12f1.Nature communications · 2025Article
- State of the art CRISPR-based strategies for cancer diagnostics and treatment.Biomarker research · 2024Review
- High-Fidelity Identification of Single Nucleotide Polymorphism by Type V CRISPR Systems.ACS sensors · 2023Article
- Improved Machine Learning-Based Model for the Classification of Off-Targets in the CRISPR/Cpf1 System.ACS omega · 2023Article
- Engineered circular guide RNAs boost CRISPR/Cas12a- and CRISPR/Cas13d-based DNA and RNA editing.Genome biology · 2023Article
- An update on CRISPR-Cas12 as a versatile tool in genome editing.Molecular biology reports · 2023Review
- Engineered Cas12a-Plus nuclease enables gene editing with enhanced activity and specificity.BMC biology · 2022Article
- Systematic decomposition of sequence determinants governing CRISPR/Cas9 specificity.Nature communications · 2022Article
- Association of clock-like mutational signature with immune checkpoint inhibitor outcome in patients with melanoma and NSCLC.Molecular therapy. Nucleic acids · 2021Article
- Rapid repair of human disease-specific single-nucleotide variants by One-SHOT genome editing.Scientific reports · 2020Article
- Allele-specific genome targeting in the development of precision medicine.Theranostics · 2020Review
- Intercellular Adhesion Molecule-1 as Target for CAR-T-Cell Therapy of Triple-Negative Breast Cancer.Frontiers in immunology · 2020Article
- Molecular mechanisms, off-target activities, and clinical potentials of genome editing systems.Clinical and translational medicine · 2020Review
- Selective targeting of the oncogenicTheranostics · 2020Article
- A Blue Light-Inducible CRISPR-Cas9 System for Inhibiting Progression of Melanoma Cells.Frontiers in molecular biosciences · 2020Article
Corrections and comments
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8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BRAF-V600E (1799T > A) is one of the most frequently reported driver mutations in multiple types of cancers, and patients with such mutations could benefit from selectively inactivating the mutant allele. Near this mutation site, there are two TTTN and one NGG protospacer-adjacent motifs (PAMs) for Cpf1 and Cas9 CRISPR nucleases, respectively. The 1799T > A substitution also leads to the occurrence of a novel NGNG PAM for the EQR variant of Cas9. We examined the editing efficacy and selectivity of Cpf1, Cas9, and EQR variant to this mutation site. Only Cpf1 demonstrated robust activity to induce specific disruption of only mutant BRAF, not wild-type sequence. Cas9 recognized and cut both normal and mutant alleles, and no obvious gene editing events were observed using EQR variant. Our results support the potential applicability of Cpf1 in precision medicine through highly specific inactivation of many other gain-of-function mutations.
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