
Ordering Information
| Product Name | Catalog # | UNIT | Price | Qty | FAVORITES | |
FMR1 Double Nickase Plasmid (h) | sc-401919-NIC | 20 µg | $410.00 | |||
FMR1 Double Nickase Plasmid (h2) | sc-401919-NIC-2 | 20 µg | $410.00 |
FMR1 encodes fragile X mental retardation protein (FMRP), an RNA-binding protein that regulates mRNA transport, stability, and translation, particularly at neuronal synapses. FMRP associates with polyribosomes and ribonucleoprotein granules to modulate activity-dependent local protein synthesis, supporting synaptic development, plasticity, and dendritic spine maturation. Through control of signaling-linked translational programs, FMR1 intersects with pathways involved in synaptic function and neuronal differentiation. Loss or dysregulation of FMR1 expression is strongly associated with fragile X–related neurodevelopmental phenotypes, making it a key target for mechanistic studies of RNA metabolism and synaptic biology.
FMR1 Double Nickase Plasmid (h) consists of a matched pair of plasmids engineered for high-specificity editing of the FMR1 locus in human cell lines. Each plasmid expresses a Cas9 D10A nickase and a distinct sgRNA targeting opposite DNA strands within FMR1. 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 FMR1 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 FMR1-disrupted clones.
For Research Use Only. Not Intended for Diagnostic or Therapeutic Use.