RNA-guided gene activation by CRISPR-Cas9-based transcription factors
Open accessAbstract
Technologies for engineering synthetic transcription factors have enabled many advances in medical and scientific research. In contrast to existing methods based on engineering of DNA-binding proteins, we created a Cas9-based transactivator that is targeted to DNA sequences by guide RNA molecules. Coexpression of this transactivator and combinations of guide RNAs in human cells induced specific expression of endogenous target genes, demonstrating a simple and versatile approach for RNA-guided gene activation.
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Because of the relatively large size of 4277 bp of dCas9-VPR cDNA, the authors used a dual rAAV system to express the dCas9-VPR and gRNA expression cassettes.
“Because of the relatively large size of 4277 bp of dCas9-VPR cDNA, the authors used a dual rAAV system to express the dCas9-VPR and gRNA expression cassettes [223,224].”
From Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays (2020) · cites this paper as [223]
The paper’s Online Methods (Plasmids; Cell culture and transfection, page unavailable) describe a dCas9-VP64 construct and transfection of plasmids encoding dCas9-VP64 and gRNAs, not a dual rAAV system expressing dCas9-VPR. Our copy does not give a 4277-bp dCas9-VPR cDNA size or attribute the delivery method to that size. (LLM, conf=0.88)
Checked by a reasoning model — may be wrong.
Experimental — accuracy not yet measuredCRISPRi and CRISPRa technologies utilize catalytically inactive Cas9 (dCas9) fused to either a transcriptional repressor (e.g., KRAB) or a transcriptional activator (e.g., VPR).
“For example, CRISPRi (interference)23–25 and CRISPRa (activation)26–30 technologies utilize catalytically inactive Cas9 (dCas9) fused to either a transcriptional repressor (e.g., KRAB, Krüppel associated box) or a transcriptional activator (e.g., VPR, VP64-p65-Rta).”
From Large-scale CRISPR screening in primary human 3D gastric organoids enables comprehensive dissection of gene-drug interactions (2025) · cites this paper as [27]
The paper’s Abstract describes a CRISPR activation system made by fusing catalytically inactive Cas9 to the VP64 transactivation domain. It does not describe the cited VPR activator; its mention of gene silencing concerns steric hindrance, not a dCas9–KRAB repressor. No page number is provided in our copy. (LLM, conf=0.88)
Experimental — accuracy not yet measured
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- Authors
- Perez-Pinera, P., Kocak, D. D., Vockley, C. M., Adler, A. F., Kabadi, A. M., Polstein, L. R., Thakore, P. I., Glass, K. A., Ousterout, D. G., Leong, K. W., Guilak, F., Crawford, G. E., Reddy, T. E., & Gersbach, C. A.
- Affiliations
- Duke University (US)
- Corresponding author
- Gersbach, Charles A
- ORCID
- Venue
- Nature Methods
- Published
- 2013-07-25
- Volume
- 10
- Issue
- 10
- Pages
- 973-976
- ISSN
- 1548-7091 (ISSN-L)
- 1548-7105
- PMC
- PMC3911785
- OpenAlex
- W2061650108
- Cited by
- 1,328
- References
- 31
- Citations by year
- 2026: 48 · 2025: 71 · 2024: 95 · 2023: 85 · 2022: 115 · 2021: 120 · 2020: 125 · 2019: 125 · 2018: 131 · 2017: 109 · 2016: 133 · 2015: 85 · 2014: 71 · 2013: 12
- FWCI
- 44.91
- Type
- journal article
- Language
- en
- Open access
- green
- europepmc.org
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Methods (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institutes of Health (1dp2od008586-01, 5u54hg004563-04)
- National Heart, Lung, and Blood Institute (R01HL109442, HL109442)
- National Human Genome Research Institute (U54‑HG004563)
- National Institute of General Medical Sciences (T32GM008555)
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (R03AR061042, R01AR48852, R01AR048852)
- National Institute of Biomedical Imaging and Bioengineering (R21EB015300, EB 015300)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Genomics and Chromatin Dynamics
- Keywords
- CRISPR/Cas9, synthetic transcription factors, transcriptional activator, guide RNA, endogenous gene expression, gene expression regulation, human cells
- MeSH terms
- Humans, Ribonucleases, Transcription Factors, Protein Engineering, Transcriptional Activation, RNA Editing, Interleukin 1 Receptor Antagonist Protein, HEK293 Cells, RNA, Small Untranslated, High-Throughput Nucleotide Sequencing, Clustered Regularly Interspaced Short Palindromic Repeats
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