ArticleMolecular therapy. Nucleic acids2020
A Small Molecule-Controlled Cas9 Repressible System.
Article in Molecular therapy. Nucleic acids, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
16 citing papers in PubMed, 35 citations in OpenAlex.
- Bio-orthogonal chemistry-based strategy to Turn-OFF CRISPR-Cas9 activity in solution and live cells.NAR molecular medicine · 2026Article
- Controlling CRISPR-Cas9 genome editing in human cells using a molecular glue degrader.Molecular therapy. Nucleic acids · 2025Article
- Reversible RNA Acylation Using Bio-Orthogonal Chemistry Enables Temporal Control of CRISPR-Cas9 Nuclease Activity.ACS chemical biology · 2024Article
- Degron tagging for rapid protein degradation in mice.Disease models & mechanisms · 2024Article
- An engineered ligand-responsive Csy4 endoribonuclease controls transgene expression from Sendai virus vectors.Journal of biological engineering · 2024Article
- Fine-Tuning the Epigenetic Landscape: Chemical Modulation of Epigenome Editors.Methods in molecular biology (Clifton, N.J.) · 2024Review
- Molecular Marvels: Small Molecules Paving the Way for Enhanced Gene Therapy.Pharmaceuticals (Basel, Switzerland) · 2023Review
- A degron system targeting endogenous PD-1 inhibits the growth of tumor cells in mice.NAR cancer · 2022Article
- Controlling CRISPR with small molecule regulation for somatic cell genome editing.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Review
- Advance trends in targeting homology-directed repair for accurate gene editing: An inclusive review of small molecules and modified CRISPR-Cas9 systems.BioImpacts : BI · 2022Review
- Small nucleic acids and the path to the clinic for anti-CRISPR.Biochemical pharmacology · 2021Review
- Review
- Approach for in vivo delivery of CRISPR/Cas system: a recent update and future prospect.Cellular and molecular life sciences : CMLS · 2021Review
- Tailoring Cardiac Synthetic Transcriptional Modulation Towards Precision Medicine.Frontiers in cardiovascular medicine · 2021Review
- Targeted protein degradation as a powerful research tool in basic biology and drug target discovery.Nature structural & molecular biology · 2020Review
- Article
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
Abstract
CRISPR-Cas9 has been developed into a powerful molecular tool for genome engineering, and it has revolutionized the field of biomedical research. Despite the tremendous potential of CRISPR-Cas9 in biomedical research, precise control of CRISPR-Cas9 over the dose and exposure time is important to expand its applications. In this study, we fused Cas9 with a peptide termed small molecule-assisted shut-off (SMASh) consisting of a protease domain and a degron domain derived from hepatitis C virus (HCV). The presence of SMASh allows tight control of the Cas9 stability via a clinically approved HCV protease inhibitor asunaprevir (ASV). We showed that the engineered Cas9 responded to ASV administration and rapidly degraded in a dose- and time-dependent manner. Cas9 degradation was reversible upon ASV removal that restored the gene editing activity. We also showed that limiting the level of Cas9 in cells increased the specificity of gene editing. The SMASh tag therefore provides an effective tool to control Cas9 stability, allowing an improvement in the accuracy, safety, and versatility of the CRISPR-Cas9 system for genome editing and gene regulation studies.
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.