ArticleCell stem cell2016
CRISPR Interference Efficiently Induces Specific and Reversible Gene Silencing in Human iPSCs.
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Article in Cell stem cell, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 312 papers.
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312citing papers in PubMed
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312 citing papers in PubMed, 534 citations in OpenAlex.
- CRISPR in clinical oncology: translational advances from molecular diagnostics to therapeutics.Nature reviews. Clinical oncology · 2026Review
- Elucidating the role of DEAF1 in neurodevelopment and shared molecular pathways in high-risk autism genes using cortical organoids.Science advances · 2026Article
- Patient-Derived Organoid-Based CRISPR Screens in Cancer Research: Applications, Advances, and Challenges.Cancer medicine · 2026Review
- Integrative analysis of drug-gene signatures in human pluripotent stem cells reveals prazosin as a novel SQSTM1 regulator for ALS therapeutics.Stem cell reports · 2026Article
- Arrayed single-gene perturbations identify drivers of human anterior neural tube closure.eLife · 2026Article
- Human pancreatic progenitor organoids define genetic and epigenetic barriers to early PDAC transformation.Developmental cell · 2026Article
- A tunable Cas12a platform for single-cell perturbation screening and CRISPRi.Nature communications · 2026Article
- Attenuation of Triple Selection Markers to Enhance mAb Expression in Tyrosine-Based Selection Systems via an Inducible CRISPRi Platform.Biotechnology journal · 2026Article
- Combinatorial and Inducible CRISPRa/i Enables Canalized hiPSC Forward Programming and Iterative RefinementbioRxiv : the preprint server for biology · 2026Article
- Embedded CRISPRi Enhances Gene-Silencing Efficiency in Drosophila.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Vimentin's Journey from "Background Scaffold" to Multi-Scale Regulator of Neuronal Growth and Function: Historical, Conceptual and Epistemic Perspectives.International journal of molecular sciences · 2026Review
- Cracking the shield: oncolytic viruses versus the tumor-immune fortress.Cancer cell international · 2026Review
- Combinatorial pioneer transcription factor binding reinforces bivalent epigenetic states to preserve lineage fidelity.bioRxiv : the preprint server for biology · 2026Article
- Intermolecular 3'UTR-3'UTR interactions drive Wnt gene activation through heteromeric protein assembly.bioRxiv : the preprint server for biology · 2026Article
- Glycaemic variability underlies myocyte dysfunction and myocardial injury risk in diabetes.Nature communications · 2026Article
- Nrf1 coordinates proteasome activity and autophagy to maintain cardiac proteostasis.Communications biology · 2026Article
- CRISPR Interference to Inhibit Oncogenes for Cancer Therapy.International journal of molecular sciences · 2026Review
- Endogenous VEGF signaling acts as a guardian of human primed pluripotency.Nature communications · 2026Article
- CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges.Molecular therapy. Nucleic acids · 2026Review
- Single cell transcriptional perturbome in pluripotent stem cell models.Molecular systems biology · 2026Article
252 more citing papers are in PubMed but not listed here.
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Authors and funding
22 authors at 4 institutions in 2 countries.
Mohammad A MandegarGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA. Electronic address: mo.mandegar@gladstone.ucsf.edu.
Nathaniel HuebschGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA; Department of Pediatrics, University of California, San Francisco, San Francisco, CA 94158, USA.
Ekaterina B FrolovGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA.
Edward ShinGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA.
Annie TruongGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA.
Michael P OlveraGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA.
Amanda H ChanGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA.
Yuichiro MiyaokaGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA.
Kristin HolmesGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA.
C Ian SpencerGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA.
Luke M JudgeGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA; Department of Pediatrics, University of California, San Francisco, San Francisco, CA 94158, USA.
David E GordonDepartment of Cellular and Molecular Pharmacology, University of California, San Francisco, San Francisco, CA 94158, USA; California Institute for Quantitative Biosciences, QB3, University of California, San Francisco, San Francisco, CA 94158, USA; Gladstone Institute of Virology and Immunology, San Francisco, CA 94158, USA.
Tilde V EskildsenDepartment of Cardiovascular and Renal Research, University of Southern Denmark, 5000 Odense C, Denmark; Department of Clinical Biochemistry and Pharmacology, Odense University Hospital, 5000 Odense C, Denmark.
Jacqueline E VillaltaDepartment of Cellular and Molecular Pharmacology, University of California, San Francisco, San Francisco, CA 94158, USA; California Institute for Quantitative Biosciences, QB3, University of California, San Francisco, San Francisco, CA 94158, USA; Howard Hughes Medical Institute, University of California, San Francisco, San Francisco, CA 94158, USA; Center for RNA Systems Biology, University of California, San Francisco, San Francisco, CA 94158, USA.
Max A HorlbeckDepartment of Cellular and Molecular Pharmacology, University of California, San Francisco, San Francisco, CA 94158, USA; California Institute for Quantitative Biosciences, QB3, University of California, San Francisco, San Francisco, CA 94158, USA; Howard Hughes Medical Institute, University of California, San Francisco, San Francisco, CA 94158, USA; Center for RNA Systems Biology, University of California, San Francisco, San Francisco, CA 94158, USA.
Luke A GilbertDepartment of Cellular and Molecular Pharmacology, University of California, San Francisco, San Francisco, CA 94158, USA; California Institute for Quantitative Biosciences, QB3, University of California, San Francisco, San Francisco, CA 94158, USA; Howard Hughes Medical Institute, University of California, San Francisco, San Francisco, CA 94158, USA; Center for RNA Systems Biology, University of California, San Francisco, San Francisco, CA 94158, USA.
Nevan J KroganDepartment of Cellular and Molecular Pharmacology, University of California, San Francisco, San Francisco, CA 94158, USA; California Institute for Quantitative Biosciences, QB3, University of California, San Francisco, San Francisco, CA 94158, USA; Gladstone Institute of Virology and Immunology, San Francisco, CA 94158, USA.
Søren P SheikhDepartment of Cardiovascular and Renal Research, University of Southern Denmark, 5000 Odense C, Denmark; Department of Clinical Biochemistry and Pharmacology, Odense University Hospital, 5000 Odense C, Denmark.
Jonathan S WeissmanDepartment of Cellular and Molecular Pharmacology, University of California, San Francisco, San Francisco, CA 94158, USA; California Institute for Quantitative Biosciences, QB3, University of California, San Francisco, San Francisco, CA 94158, USA; Howard Hughes Medical Institute, University of California, San Francisco, San Francisco, CA 94158, USA; Center for RNA Systems Biology, University of California, San Francisco, San Francisco, CA 94158, USA.
Lei S QiDepartment of Bioengineering, Stanford University, Stanford, CA 94305, USA.
Po-Lin SoGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA.
Bruce R ConklinGladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA; Department of Cellular and Molecular Pharmacology, University of California, San Francisco, San Francisco, CA 94158, USA; California Institute for Quantitative Biosciences, QB3, University of California, San Francisco, San Francisco, CA 94158, USA; Department of Medicine and Cellular and Molecular Pharmacology, University of California, San Francisco, San Francisco, CA 94158, USA. Electronic address: bconklin@gladstone.ucsf.edu.
Gladstone Institutes · USQB3 · USUniversity of Southern Denmark · DKStanford University · US
Funding
Virology CoreP30AI027763 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI STEVEN Grant DEEKS · 1988 to 2026
$93.6MProgenitor Cell Biology Consortium Administrative Coordinating CenterU01HL099997 · NHLBI · UNIVERSITY OF MARYLAND BALTIMORE · PI TERRIN, MICHAEL L · 2009 to 2015
$29.9MMEDICAL SCIENTIST TRAINING PROGRAMT32GM007618 · NIGMS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI ANDERSON, MARK S · 1985 to 2020
$29.6MTranscriptional Networks During Cardiac DifferentiationP01HL089707 · NHLBI · J. DAVID GLADSTONE INSTITUTES · PI SRIVASTAVA, DEEPAK · 2008 to 2017
$20.4MSTRUCTURAL BIOLOGY TRAINING PROGRAMT32GM008284 · NIGMS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI KORTEMME, TANJA · 1988 to 2022
$10.1MThe Epigenetic Landscape of Heart DevelopmentU01HL098179 · NHLBI · J. DAVID GLADSTONE INSTITUTES · PI BOYER, LAURIE A, BRUNEAU, BENOIT GAETAN · 2009 to 2014
$10.0MInduced Pluripotent Stem Cells in the Understanding and Treatment of Heart DiseasU01HL100406 · NHLBI · J. DAVID GLADSTONE INSTITUTES · PI BRUNEAU, BENOIT GAETAN, CONKLIN, BRUCE R · 2009 to 2015
$9.4MTissue Engineering with a Modular RASSL ToolboxR01HL060664 · NHLBI · J. DAVID GLADSTONE INSTITUTES · PI CONKLIN, BRUCE R · 1998 to 2012
$7.0MTraining in Developmental Cardiovascular BiologyT32HL007544 · NHLBI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI SRIVASTAVA, DEEPAK · 1985 to 2021
$4.5MIntegrative Program in Complex Biological System (ipCBS)T32EB009383 · NIBIB · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI MARSHALL, WALLACE, WEINER, ORION D · 2009 to 2022
$3.2MHarnessing CRISPR for Targeted and Inducible Epigenomic ReprogrammingR01DA036858 · NIDA · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI LIM, WENDELL A, QI, LEI STANLEY · 2013 to 2017
$2.9MDisease Specific Cardiac Tissue ModelsR01HL108677 · NHLBI · UNIVERSITY OF CALIFORNIA BERKELEY · PI CONKLIN, BRUCE R, HEALY, KEVIN EDWARD · 2011 to 2014
$2.4MCanadian Institutes of Health Research 129844Howard Hughes Medical InstituteNHLBI NIH HHS P01 HL089707NHLBI NIH HHS P01-HL089707NHLBI NIH HHS R01 HL060664NHLBI NIH HHS R01-HL060664NHLBI NIH HHS R01 HL108677NHLBI NIH HHS R01-HL108677NHLBI NIH HHS R01 HL130533NHLBI NIH HHS R01HL130533NHLBI NIH HHS T32 HL007544NHLBI NIH HHS U01 HL098179NHLBI NIH HHS U01-HL098179NHLBI NIH HHS U01 HL099997NHLBI NIH HHS U01-HL099997NHLBI NIH HHS U01 HL100406NHLBI NIH HHS U01-HL100406NIAID NIH HHS P30 AI027763NIBIB NIH HHS T32 EB009383NICHD NIH HHS 1K12HD072222NICHD NIH HHS K12 HD072222NIDA NIH HHS R01 DA036858NIGMS NIH HHS T32 GM007618NIGMS NIH HHS T32 GM008284NIGMS NIH HHS U01-GM09614
Abstract
Developing technologies for efficient and scalable disruption of gene expression will provide powerful tools for studying gene function, developmental pathways, and disease mechanisms. Here, we develop clustered regularly interspaced short palindromic repeat interference (CRISPRi) to repress gene expression in human induced pluripotent stem cells (iPSCs). CRISPRi, in which a doxycycline-inducible deactivated Cas9 is fused to a KRAB repression domain, can specifically and reversibly inhibit gene expression in iPSCs and iPSC-derived cardiac progenitors, cardiomyocytes, and T lymphocytes. This gene repression system is tunable and has the potential to silence single alleles. Compared with CRISPR nuclease (CRISPRn), CRISPRi gene repression is more efficient and homogenous across cell populations. The CRISPRi system in iPSCs provides a powerful platform to perform genome-scale screens in a wide range of iPSC-derived cell types, dissect developmental pathways, and model disease.
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Gene SilencingClustered Regularly Interspaced Short Palindromic RepeatsHumansInduced Pluripotent Stem Cells
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