Date published: 2026-7-20

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ARSA Double Nickase Plasmid (h): sc-404921-NIC

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Datasheets
  • Target species: human
  • 20 µg of transfection-ready, purified plasmid DNA; Suitable for up to 20 transfections
  • ARSA 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
  • ARSA Double Nickase Plasmid (h) and ARSA Double Nickase Plasmid (h2) encode distinct paired gRNA designs targeting ASNA1. One or both designs may be available
  • Following transfection, gene knockout efficiency can be assayed by WB, IF or IHC using antibody: ARSA Antibody (H-7): sc-390568
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    Ordering Information

    Product NameCatalog #UNITPriceQtyFAVORITES

    ARSA Double Nickase Plasmid (h)

    sc-404921-NIC
    20 µg
    $410.00

    ARSA Double Nickase Plasmid (h2)

    sc-404921-NIC-2
    20 µg
    $410.00

    Human ARSA (arylsulfatase A) is a lysosomal sulfatase that hydrolyzes cerebroside 3-sulfate (sulfatide), supporting sphingolipid turnover and myelin lipid homeostasis. By coupling to lysosomal catabolic pathways and endolysosomal trafficking, ARSA helps regulate membrane composition and prevents sulfatide accumulation in neural and glial cells. Loss of ARSA activity is linked to disrupted lysosomal function and demyelinating pathology observed in metachromatic leukodystrophy, making ARSA a widely used node for studying lysosomal storage biology and neurodegeneration-associated lipid dysregulation. ARSA perturbation is also relevant for investigating how sphingolipid metabolites influence inflammation, cellular stress responses, and neuronal viability.

    ARSA Double Nickase Plasmid (h) consists of a matched pair of plasmids engineered for high-specificity editing of the ASNA1 locus in human cell lines. Each plasmid expresses a Cas9 D10A nickase and a distinct sgRNA targeting opposite DNA strands within ASNA1. 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 ASNA1 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 ASNA1-disrupted clones.

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