alpha-L-iduronidase Double Nickase Plasmid (h): sc-403722-NIC
- Target species: human
- 20 µg of transfection-ready, purified plasmid DNA; Suitable for up to 20 transfections
- alpha-L-iduronidase Double Nickase Plasmid (h) consists of a pair of plasmids each encoding a D10A mutated Cas9 nuclease and a target-specific 20 nt guide RNA (gRNA) designed to knockout gene expression with greater specificity than its CRISPR/Cas9 KO counterpart
- Paired gRNA sequences are offset by approximately 20 bp to allow for specific Cas9-mediated double nicking of the genomic DNA, which mimics a DSB
- One plasmid in the pair contains a puromycin-resistance gene for selection; the other plasmid in the pair contains a GFP marker to visually confirm transfection
- alpha-L-iduronidase Double Nickase Plasmid (h) and alpha-L-iduronidase Double Nickase Plasmid (h2) encode distinct paired gRNA designs targeting IDUA. One or both designs may be available
Ordering Information
| Product Name | Catalog # | UNIT | Price | Qty | FAVORITES | |
alpha-L-iduronidase Double Nickase Plasmid (h) | sc-403722-NIC | 20 µg | $410.00 | |||
alpha-L-iduronidase Double Nickase Plasmid (h2) | sc-403722-NIC-2 | 20 µg | $410.00 |
Human IDUA encodes the lysosomal hydrolase alpha-L-iduronidase, a key enzyme in glycosaminoglycan catabolism that cleaves terminal iduronic acid residues from dermatan sulfate and heparan sulfate. Proper IDUA activity supports lysosome-dependent turnover, endo-lysosomal trafficking, and cellular proteostasis by preventing accumulation of undegraded polysaccharides. Perturbation of this pathway is linked to lysosomal storage pathology and downstream effects on extracellular matrix remodeling and inflammatory signaling. IDUA is therefore widely used as a functional readout for lysosomal biology, substrate flux, and genotype–phenotype relationships in human cell models.
alpha-L-iduronidase Double Nickase Plasmid (h) consists of a matched pair of plasmids engineered for high-specificity editing of the IDUA locus in human cell lines. Each plasmid expresses a Cas9 D10A nickase and a distinct sgRNA targeting opposite DNA strands within IDUA. 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 IDUA 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 IDUA-disrupted clones.
For Research Use Only. Not Intended for Diagnostic or Therapeutic Use.