ArticleNucleic acids research2021
MiniCAFE, a CRISPR/Cas9-based compact and potent transcriptional activator, elicits gene expression in vivo.
Article in Nucleic acids research, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 27 papers.
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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.
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Who cites it
27 citing papers in PubMed, 53 citations in OpenAlex.
- TIGRa: An ultra-compact programmable activator enabling multiplexed and efficient gene regulation.Cell stem cell · 2026Article
- Simultaneous orthogonal cell engineering by a single CRISPR-Cas9 polyfunctional editor.Nature communications · 2026Article
- exoCasMINI: A T5 exonuclease fused CRISPR-Cas12f system with enhanced gene editing efficiency.iScience · 2025Article
- CRISPR-Cas technologies: Emerging tools from research to clinical application.Journal of microbiology (Seoul, Korea) · 2025Review
- hpCasMINI: An engineered hypercompact CRISPR-Cas12f system with boosted gene editing activity.Nature communications · 2025Article
- Engineered circular guide RNAs enhance miniature CRISPR/Cas12f-based gene activation and adenine base editing.Nature communications · 2025Article
- SunTag-PE: a modular prime editing system enables versatile and efficient genome editing.Communications biology · 2025Article
- Gene therapy as an emerging treatment forFundamental research · 2024Review
- Precision epigenetic editing: Technological advances, enduring challenges, and therapeutic applications.Cell chemical biology · 2024Review
- TFAM is an autophagy receptor that limits inflammation by binding to cytoplasmic mitochondrial DNA.Nature cell biology · 2024Article
- Compact engineered human mechanosensitive transactivation modules enable potent and versatile synthetic transcriptional control.Nature methods · 2023Article
- Advances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases.Biomedicines · 2023Review
- Engineered circular guide RNAs boost CRISPR/Cas12a- and CRISPR/Cas13d-based DNA and RNA editing.Genome biology · 2023Article
- Phase-separated DropCRISPRa platform for efficient gene activation in mammalian cells and mice.Nucleic acids research · 2023Article
- The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss.Cell & bioscience · 2023Review
- Profiling the impact of the promoters on CRISPR-Cas12a system in human cells.Cellular & molecular biology letters · 2023Article
- Review
- Targeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery.Experimental & molecular medicine · 2023Article
- Comparison of DNA targeting CRISPR editors in human cells.Cell & bioscience · 2023Article
- Applications of CRISPR screening to lung cancer treatment.Frontiers in cell and developmental biology · 2023Review
Corrections and comments
- Erratum issued
Authors and funding
19 authors at 5 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
CRISPR-mediated gene activation (CRISPRa) is a promising therapeutic gene editing strategy without inducing DNA double-strand breaks (DSBs). However, in vivo implementation of these CRISPRa systems remains a challenge. Here, we report a compact and robust miniCas9 activator (termed miniCAFE) for in vivo activation of endogenous target genes. The system relies on recruitment of an engineered minimal nuclease-null Cas9 from Campylobacter jejuni and potent transcriptional activators to a target locus by a single guide RNA. It enables robust gene activation in human cells even with a single DNA copy and is able to promote lifespan of Caenorhabditis elegans through activation of longevity-regulating genes. As proof-of-concept, delivered within an all-in-one adeno-associated virus (AAV), miniCAFE can activate Fgf21 expression in the liver and regulate energy metabolism in adult mice. Thus, miniCAFE holds great therapeutic potential against human diseases.
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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.