
Ordering Information
| Product Name | Catalog # | UNIT | Price | Qty | FAVORITES | |
RPGR CRISPR Activation Plasmid (h) | sc-404441-ACT | 20 µg | $397.00 |
Human RPGR (retinitis pigmentosa GTPase regulator) localizes predominantly to the connecting cilium of photoreceptors and to centrosome/basal body–associated compartments, where it supports ciliary protein trafficking and microtubule-based transport. Through interactions with ciliary and intraflagellar transport machinery, RPGR helps maintain outer segment integrity and photoreceptor homeostasis. Disruption of RPGR-dependent ciliary processes is strongly linked to retinal degeneration phenotypes, including X-linked retinitis pigmentosa and related inherited retinal dystrophies, highlighting its relevance to sensory cilia biology.
RPGR CRISPR Activation Plasmid (h) provides a targeted, non-destructive approach to upregulating endogenous RPGR expression without altering the underlying DNA sequence.
RPGR CRISPR Activation Plasmid (h) is a three-plasmid synergistic activation mediator (SAM) system engineered for highly efficient, site-specific transcriptional upregulation of the RPGR locus in human cell lines. The system is built around a catalytically inactive Cas9 (dCas9) carrying two inactivating mutations (D10A and N863A) that eliminate nuclease activity while preserving DNA binding. This dCas9 is fused to VP64, a potent transcriptional activator, and is co-expressed with a blasticidin resistance gene for selection. The second plasmid encodes the MS2-p65-HSF1 fusion protein, a secondary activator complex that works in concert with dCas9-VP64, alongside a hygromycin resistance gene. The third plasmid encodes a target-specific 20 nt sgRNA fused to two MS2 RNA aptamers that recruit the MS2-p65-HSF1 complex to the activation site, accompanied by a puromycin resistance gene. The three plasmids are delivered at a 1:1:1 mass ratio for balanced expression of all system components.
Once assembled at the target locus, the SAM complex binds within approximately 200 bp upstream of the RPGR transcriptional start site, where VP64, p65, and HSF1 act in concert to recruit transcriptional machinery and drive upregulation of endogenous RPGR expression. Unlike nuclease-active Cas9, dCas9 does not introduce double-strand breaks or modify the genomic sequence, preserving the native RPGR locus and enabling the study of RPGR-dependent transcriptional responses at the endogenous locus, making it a valuable tool for functional studies, target gene identification, and the modeling of RPGR pathway restoration in tumor cells with silenced or reduced RPGR expression.
For Research Use Only. Not Intended for Diagnostic or Therapeutic Use.