ArticleMolecular therapy : the journal of the American Society of Gene Therapy2020
Doxycycline-Dependent Self-Inactivation of CRISPR-Cas9 to Temporally Regulate On- and Off-Target Editing.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
22 citing papers in PubMed, 25 citations in OpenAlex.
- STRAIGHT-IN Dual: a platform for dual single-copy integrations of DNA payloads and gene circuits into human induced pluripotent stem cells.Nature biomedical engineering · 2026Article
- Review
- Mechanisms of and mitigating strategies for cellular immune responses to CRISPR-associated nucleases in genome editing therapy.Frontiers in medicine · 2026Review
- Controlling CRISPR-Cas9 genome editing in human cells using a molecular glue degrader.Molecular therapy. Nucleic acids · 2025Article
- STRAIGHT-IN Dual: a platform for dual, single-copy integrations of DNA payloads and gene circuits into human induced pluripotent stem cell.bioRxiv : the preprint server for biology · 2025Article
- Novel epigenetic molecular therapies for imprinting disorders.Molecular psychiatry · 2023Review
- Not all (cells) who wander are lost: Upstream migration as a pervasive mode of amoeboid cell motility.Frontiers in cell and developmental biology · 2023Review
- Small Molecules for Enhancing the Precision and Safety of Genome Editing.Molecules (Basel, Switzerland) · 2022Review
- CRISPR-AsCas12a Efficiently Corrects aThe CRISPR journal · 2022Article
- Forward Genetic Screens of Human Glycosylation Pathways Using the GlycoGene CRISPR Library.Current protocols · 2022Article
- Controlling CRISPR with small molecule regulation for somatic cell genome editing.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Review
- Evaluation of twoMolecular therapy. Methods & clinical development · 2021Article
- Cas9-directed immune tolerance in humans-a model to evaluate regulatory T cells in gene therapy?Gene therapy · 2021Review
- A new mouse SNP genotyping assay for speed congenics: combining flexibility, affordability, and power.BMC genomics · 2021Article
- A GlycoGene CRISPR-Cas9 lentiviral library to study lectin binding and human glycan biosynthesis pathways.Glycobiology · 2021Article
- A systems-based framework to computationally describe putative transcription factors and signaling pathways regulating glycan biosynthesis.Beilstein journal of organic chemistry · 2021Article
- Gene Editing by Extracellular Vesicles.International journal of molecular sciences · 2020Review
- New Directions in Pulmonary Gene Therapy.Human gene therapy · 2020Review
- Treating Cystic Fibrosis with mRNA and CRISPR.Human gene therapy · 2020Review
- Transcriptional repression of PTEN in neural cells using CRISPR/dCas9 epigenetic editing.Scientific reports · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 2 institutions in 1 country.
Funding
Abstract
Exome and deep sequencing of cells treated with a panel of lentiviral guide RNA demonstrate that both on- and off-target editing proceed in a time-dependent manner. Thus, methods to temporally control Cas9 activity would be beneficial. To address this need, we describe a "self-inactivating CRISPR (SiC)" system consisting of a single guide RNA that deactivates the Streptococcus pyogenes Cas9 nuclease in a doxycycline-dependent manner. This enables defined, temporal control of Cas9 activity in any cell type and also in vivo. Results show that SiC may enable a reduction in off-target editing, with less effect on on-target editing rates. This tool facilitates diverse applications including (1) the timed regulation of genetic knockouts in hard-to-transfect cells using lentivirus, including human leukocytes for the identification of glycogenes regulating leukocyte-endothelial cell adhesion; (2) genome-wide lentiviral sgRNA (single guide RNA) library applications where Cas9 activity is ablated after allowing pre-determined editing times. Thus, stable knockout cell pools are created for functional screens; and (3) temporal control of Cas9-mediated editing of myeloid and lymphoid cells in vivo, both in mouse peripheral blood and bone marrow. Overall, SiC enables temporal control of gene editing and may be applied in diverse application including studies that aim to reduce off-target genome editing.
Indexed as
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.