ArticleMolecular therapy : the journal of the American Society of Gene Therapy2018
Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 papers, 1 of them a synthesis that pooled it.
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Who cites it
38 citing papers in PubMed, 1 synthesis or guideline pooled it.
- RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges.Pharmacological reviews · 2020Pooled it
- Cas12a chRDNA-mediatedMolecular therapy. Nucleic acids · 2026Article
- Article
- Identification of thermotolerant non-canonical PAMs for robust one-pot CRISPR-Cas12a detection.Nature communications · 2026Article
- Steric regulation of CRISPR/Cas12a trans-cleavage kinetics via split-activator extensions.Nucleic acids research · 2026Article
- RNA chemistry and therapeutics.Nature reviews. Drug discovery · 2025Review
- Regulating cleavage activity and enabling microRNA detection with split sgRNA in Cas12b.Nature communications · 2025Article
- Recent advances in CRISPR-based single-nucleotide fidelity diagnostics.Communications medicine · 2025Review
- Enhancement of CRISPR-Cas12a system through universal circular RNA design.Cell reports methods · 2025Article
- Advances in actinobacteria-based bioremediation: mechanistic insights, genetic regulation, and emerging technologies.Biodegradation · 2025Review
- Chemical engineering of CRISPR-Cas systems for therapeutic application.Nature reviews. Drug discovery · 2025Review
- Expanding Cas12a Activity Control with an RNA G-Quadruplex at the 5' end of CRISPR RNA.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Direct repeat region 3' end modifications regulate Cas12a activity and expand its applications.Nucleic acids research · 2025Article
- Chemical Modifications in Nucleic Acid Therapeutics.Methods in molecular biology (Clifton, N.J.) · 2025Review
- Allele-specific CRISPR-Cas9 editing of dominant epidermolysis bullosa simplex in human epidermal stem cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Harnessing non-Watson-Crick's base pairing to enhance CRISPR effectors cleavage activities and enable gene editing in mammalian cells.Proceedings of the National Academy of Sciences of the United States of America · 2024Article
- Unmodificated stepless regulation of CRISPR/Cas12a multi-performance.Nucleic acids research · 2023Article
- CRISPR Assays for Disease Diagnosis: Progress to and Barriers Remaining for Clinical Applications.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2023Review
- Engineered circular guide RNAs boost CRISPR/Cas12a- and CRISPR/Cas13d-based DNA and RNA editing.Genome biology · 2023Article
- Regulation of the CRISPR-Cas12a system by methylation and demethylation of guide RNA.Chemical science · 2023Article
Corrections and comments
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Authors and funding
5 authors.
Funding
Abstract
CRISPR-based gene editing is a powerful technology for engineering mammalian genomes. It holds the potential as a therapeutic, although much-needed in vivo delivery systems have yet to be established. Here, using the Cpf1-crRNA (CRISPR RNA) crystal structure as a guide, we synthesized a series of systematically truncated and chemically modified crRNAs, and identify positions that are amenable to modification while retaining gene-editing activity. Modified crRNAs were designed with the same modifications that provide protection against nucleases and enable wide distribution in vivo. We show crRNAs with chemically modified terminal nucleotides are exonuclease resistant while retaining gene-editing activity. Chemically modified or DNA-substituted nucleotides at select positions and up to 70% of the crRNA DNA specificity region are also well tolerated. In addition, gene-editing activity is maintained with phosphorothioate backbone substitutions in the crRNA DNA specificity region. Finally, we demonstrate that 42-mer synthetic crRNAs from the similar CRISPR-Cas9 system are taken up by cells, an attractive property for in vivo delivery. Our study is the first to show that chemically modified crRNAs of the CRISPR-Cpf1 system can functionally replace and mediate comparable gene editing to the natural crRNA, which holds the potential for enhancing both viral- and non-viral-mediated in vivo gene editing.
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Registered trials
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