matrin-3 CRISPR Activation Plasmid (h): sc-418085-ACT

MATR3 encodes matrin-3, a nuclear matrix RNA/DNA-binding protein that contributes to nuclear architecture and coordinates gene expression through interactions with chromatin and ribonucleoprotein complexes. Matrin-3 participates in RNA splicing, mRNA stabilization, and nuclear retention, linking it to regulation of transcript processing and stress-responsive gene programs. Its functional network intersects with pathways governing RNA metabolism and nucleocytoplasmic organization, processes that are frequently perturbed in neurodegeneration. Dysregulated MATR3 activity and pathogenic variants have been associated with motor neuron disease and related neuromuscular phenotypes, supporting its use as a mechanistic node for studying RNA-binding protein biology.

matrin-3 CRISPR Activation Plasmid (h) provides a targeted, non-destructive approach to upregulating endogenous MATR3 expression without altering the underlying DNA sequence.

matrin-3 CRISPR Activation Plasmid (h) is a three-plasmid synergistic activation mediator (SAM) system engineered for highly efficient, site-specific transcriptional upregulation of the MATR3 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 MATR3 transcriptional start site, where VP64, p65, and HSF1 act in concert to recruit transcriptional machinery and drive upregulation of endogenous matrin-3 expression. Unlike nuclease-active Cas9, dCas9 does not introduce double-strand breaks or modify the genomic sequence, preserving the native MATR3 locus and enabling the study of matrin-3-dependent transcriptional responses at the endogenous locus, making it a valuable tool for functional studies, target gene identification, and the modeling of matrin-3 pathway restoration in tumor cells with silenced or reduced MATR3 expression.

For Research Use Only. Not Intended for Diagnostic or Therapeutic Use.