
Ordering Information
| Product Name | Catalog # | UNIT | Price | Qty | FAVORITES | |
NPC1 Double Nickase Plasmid (h) | sc-403252-NIC | 20 µg | $410.00 | |||
NPC1 Double Nickase Plasmid (h2) | sc-403252-NIC-2 | 20 µg | $410.00 |
NPC1 encodes Niemann-Pick disease type C1 protein, a late endosomal/lysosomal membrane transporter that coordinates intracellular cholesterol and glycolipid trafficking. By partnering with NPC2 and regulating sterol egress from lysosomes to other membranes, NPC1 supports lipid homeostasis, membrane composition, and downstream signaling linked to endosome–lysosome dynamics and autophagy. Disruption of NPC1 function causes lysosomal lipid accumulation and cellular stress phenotypes, and variants are associated with Niemann-Pick disease type C and broader neurodegeneration- and metabolism-related research. NPC1 is therefore widely studied in pathways governing lysosomal function, organelle contact sites, and cholesterol-dependent signaling.
NPC1 Double Nickase Plasmid (h) consists of a matched pair of plasmids engineered for high-specificity editing of the NPC1 locus in human cell lines. Each plasmid expresses a Cas9 D10A nickase and a distinct sgRNA targeting opposite DNA strands within NPC1. When directed to adjacent sites on opposite DNA strands, the two nickases generate offset single-strand nicks that together produce a staggered double-strand break, requiring coordinated on-target activity from both guides. The resulting DNA break is resolved by endogenous cellular repair pathways, most commonly through non-homologous end joining (NHEJ), leading to insertions or deletions that disrupt NPC1 function. By requiring dual sgRNA engagement at the target locus, the double nicking approach enhances editing specificity and provides a complementary CRISPR strategy for applications where additional control over targeting precision is desired.
To support efficient identification of edited cells, one plasmid encodes GFP for fluorescent visualization of transfected populations, while the companion plasmid carries a puromycin resistance gene for antibiotic selection. Together, these features support efficient enrichment of co-transfected populations and simplify the validation of NPC1-disrupted clones.
For Research Use Only. Not Intended for Diagnostic or Therapeutic Use.