
Ordering Information
| Product Name | Catalog # | UNIT | Price | Qty | FAVORITES | |
HEXA Double Nickase Plasmid (h) | sc-405523-NIC | 20 µg | $410.00 | |||
HEXA Double Nickase Plasmid (h2) | sc-405523-NIC-2 | 20 µg | $410.00 |
HEXA encodes the α-subunit of β-hexosaminidase A, a lysosomal glycosidase that partners with the β-subunit (HEXB) to catalyze stepwise degradation of GM2 ganglioside and related glycoconjugates. This activity is central to lysosome-mediated catabolism and lipid homeostasis, linking HEXA function to endolysosomal trafficking, autophagy–lysosome crosstalk, and cellular stress responses triggered by substrate accumulation. Loss-of-function variants in HEXA are associated with GM2 gangliosidosis (Tay–Sachs disease), a model system for investigating how lysosomal dysfunction perturbs neuronal physiology, membrane composition, and proteostasis. As a research target, HEXA supports studies of lysosomal biology, glycosphingolipid metabolism, and genotype–phenotype relationships in human cell models.
HEXA Double Nickase Plasmid (h) consists of a matched pair of plasmids engineered for high-specificity editing of the HEXA locus in human cell lines. Each plasmid expresses a Cas9 D10A nickase and a distinct sgRNA targeting opposite DNA strands within HEXA. 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 HEXA 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 HEXA-disrupted clones.
For Research Use Only. Not Intended for Diagnostic or Therapeutic Use.