ReviewNature protocols2022
dCas9-VPR-mediated transcriptional activation of functionally equivalent genes for gene therapy.
Review in Nature protocols, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed.
- Compact transcriptional activators: Therapeutic gene regulation in ocular disease.Neural regeneration research · 2026Article
- CRISPR-Cas9: Genome Engineering and Future Vaccine Applications.Molecular biotechnology · 2026Review
- High-content CRISPR activation screens identify synthetically lethal RNA-based mechanisms to sensitize cancer cells to targeted T cell cytotoxicity.Nature genetics · 2026Article
- Dual AAV vectors for efficient delivery of large transgenes.Nature protocols · 2026Review
- Epigenetic editing: from concept to clinic.Nature reviews. Drug discovery · 2026Review
- A novel minimally humanized mouse model of aniridia for preclinical evaluation of CRISPR gene editing strategies.Frontiers in genome editing · 2026Article
- Screening and Identification of Hnf1ba-International journal of molecular sciences · 2025Article
- CRISPR/Cas-Mediated Gene Activation as a Versatile Tool for Treatment of Inherited Retinal Dystrophies.Advances in experimental medicine and biology · 2025Review
- Regulation of CAR transgene expression to design semiautonomous CAR-T.Molecular therapy. Oncology · 2024Review
- Activating UCHL1 through the CRISPR activation system promotes cartilage differentiation mediated by HIF-1α/SOX9.Journal of cellular and molecular medicine · 2024Article
- Logical regulation of endogenous gene expression using programmable, multi-input processing CRISPR guide RNAs.Nucleic acids research · 2024Article
- Optogenetics and Targeted Gene Therapy for Retinal Diseases: Unravelling the Fundamentals, Applications, and Future Perspectives.Journal of clinical medicine · 2024Review
- CRISPR-assisted transcription activation by phase-separation proteins.Protein & cell · 2023Article
- CRISPR/Cas9 Landscape: Current State and Future Perspectives.International journal of molecular sciences · 2023Review
- mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy.Nature communications · 2023Article
- CRISPR-Cas9 Direct Fusions for Improved Genome Editing via Enhanced Homologous Recombination.International journal of molecular sciences · 2023Review
- Novel epigenetic molecular therapies for imprinting disorders.Molecular psychiatry · 2023Review
Corrections and comments
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Many disease-causing genes possess functionally equivalent counterparts, which are often expressed in distinct cell types. An attractive gene therapy approach for inherited disorders caused by mutations in such genes is to transcriptionally activate the appropriate counterpart(s) to compensate for the missing gene function. This approach offers key advantages over conventional gene therapies because it is mutation- and gene size-independent. Here, we describe a protocol for the design, execution and evaluation of such gene therapies using dCas9-VPR. We offer guidelines on how to identify functionally equivalent genes, design and clone single guide RNAs and evaluate transcriptional activation in vitro. Moreover, focusing on inherited retinal diseases, we provide a detailed protocol on how to apply this strategy in mice using dual recombinant adeno-associated virus vectors and how to evaluate its functionality and off-target effects in the target tissue. This strategy is in principle applicable to all organisms that possess functionally equivalent genes suitable for transcriptional activation and addresses pivotal unmet needs in gene therapy with high translational potential. The protocol can be completed in 15-20 weeks.
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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.