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dCas9-VPR
- Development stage
- Preclinical
- Lead developer
- University of Florida
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Recombinant Proteins and Enzymes
- Administration
- Parenteral
01
Overview
dCas9-VPR is a CRISPR-mediated gene activation (CRISPRa) system consisting of a catalytically inactive "dead" Cas9 (dCas9) protein fused to a potent tripartite transcriptional activator domain comprising VP64, p65, and Rta (VPR). Unlike traditional CRISPR-Cas9, which induces double-strand breaks to edit the genome, dCas9-VPR is guided by single guide RNAs (sgRNAs) to specific promoter or enhancer regions where it recruits the cellular transcriptional machinery to upregulate the expression of endogenous genes. In the context of regenerative medicine, this technology is being utilized to engineer human pluripotent stem cells (hPSCs) to induce the maturation of stem cell-derived beta-cells (sBCs). By simultaneously activating specific transcription factor networks that are typically low or absent in sBCs, researchers aim to create mature, functional beta-cells for the treatment of Type 1 Diabetes.
- Other names
- dCas9-VP64-p65-RtadCas-9-VP64-p65-RtadCas 9-VP64-p65-RtaCRISPRa dCas9-VPR
02
Targets
Protospacer (Genomic DNA target sequence)
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