ABCA13 CRISPR Activation Plasmid (h): sc-407004-ACT

ABCA13 encodes a large ATP-binding cassette (ABC) transporter implicated in ATP-dependent lipid translocation across cellular membranes, supporting membrane organization and intracellular trafficking. As a member of the ABCA subfamily, ABCA13 is linked to lipid and cholesterol handling processes that can influence vesicular transport, endosomal–lysosomal dynamics, and epithelial cell homeostasis. Altered ABCA13 expression has been reported in pulmonary biology and has been associated with cancer- and neuropsychiatric-related datasets, motivating studies of how transporter-driven lipid remodeling affects signaling and cellular differentiation. These features make ABCA13 a useful target for investigating lipid transport pathways, membrane composition-dependent signaling, and disease-relevant transcriptional programs in human cell models.

ABCA13 CRISPR Activation Plasmid (h) provides a targeted, non-destructive approach to upregulating endogenous ABCA13 expression without altering the underlying DNA sequence.

ABCA13 CRISPR Activation Plasmid (h) is a three-plasmid synergistic activation mediator (SAM) system engineered for highly efficient, site-specific transcriptional upregulation of the ABCA13 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 ABCA13 transcriptional start site, where VP64, p65, and HSF1 act in concert to recruit transcriptional machinery and drive upregulation of endogenous ABCA13 expression. Unlike nuclease-active Cas9, dCas9 does not introduce double-strand breaks or modify the genomic sequence, preserving the native ABCA13 locus and enabling the study of ABCA13-dependent transcriptional responses at the endogenous locus, making it a valuable tool for functional studies, target gene identification, and the modeling of ABCA13 pathway restoration in tumor cells with silenced or reduced ABCA13 expression.

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