



Ordering Information
| Product Name | Catalog # | UNIT | Price | Qty | FAVORITES | |
ABCA4 Double Nickase Plasmid (h) | sc-401830-NIC | 20 µg | $410.00 | |||
ABCA4 Double Nickase Plasmid (h2) | sc-401830-NIC-2 | 20 µg | $410.00 |
ABCA4 encodes an ATP-binding cassette transporter predominantly localized to photoreceptor outer segment disc membranes, where it functions as an ATP-dependent flippase for retinoid-derived phospholipids. By facilitating clearance of N-retinylidene-phosphatidylethanolamine and related bisretinoid precursors, ABCA4 supports the visual (retinoid) cycle and protects cells from photooxidative stress. Disruption of ABCA4 activity perturbs retinaldehyde handling and promotes accumulation of toxic lipofuscin fluorophores within the retinal pigment epithelium. Variants in ABCA4 are strongly associated with inherited retinal degenerations, making it a key target for mechanistic studies of photoreceptor metabolism and cellular stress pathways.
ABCA4 Double Nickase Plasmid (h) consists of a matched pair of plasmids engineered for high-specificity editing of the ABCA4 locus in human cell lines. Each plasmid expresses a Cas9 D10A nickase and a distinct sgRNA targeting opposite DNA strands within ABCA4. 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 ABCA4 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 ABCA4-disrupted clones.
For Research Use Only. Not Intended for Diagnostic or Therapeutic Use.