
Ordering Information
| Product Name | Catalog # | UNIT | Price | Qty | FAVORITES | |
MBNL1 Double Nickase Plasmid (h) | sc-402194-NIC | 20 µg | $410.00 | |||
MBNL1 Double Nickase Plasmid (h2) | sc-402194-NIC-2 | 20 µg | $410.00 |
MBNL1 (muscleblind-like splicing regulator 1) encodes an RNA-binding protein that recognizes YGCY motifs in pre-mRNAs to control alternative splicing, mRNA localization, and stability. It coordinates post-transcriptional gene regulation programs important for myogenesis, neuronal differentiation, and maintenance of tissue-specific isoform expression. MBNL1 participates in RNA processing and spliceosome-related pathways and helps establish developmental splicing transitions across many transcripts. Dysregulation or functional sequestration of MBNL1 is strongly linked to aberrant splicing signatures observed in myotonic dystrophy and other neuromuscular phenotypes, making it a key node for mechanistic studies of RNA misprocessing.
MBNL1 Double Nickase Plasmid (h) consists of a matched pair of plasmids engineered for high-specificity editing of the MBNL1 locus in human cell lines. Each plasmid expresses a Cas9 D10A nickase and a distinct sgRNA targeting opposite DNA strands within MBNL1. 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 MBNL1 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 MBNL1-disrupted clones.
For Research Use Only. Not Intended for Diagnostic or Therapeutic Use.