Nusinersen
Product Specifications
CAS Number
1258984-36-9
Target
DNA/RNA Synthesis
Applications
Cell Cycle
Additionnal Information
Nusinersen is an 18-mer, 2′-O- (2-methoxyethyl) phosphorothioate antisense oligonucleotide used for treating spinal muscular atrophy (SMA) . It binds specifically to the intronic splice silencer N1 (ISS-N1) site in intron 7 of SMN2 pre-mRNA, blocking the binding of splicing repressors hnRNP A1/A2. This promotes inclusion of exon 7 during splicing, restoring full-length SMN protein production in SMA patients.
Frequently Asked Questions
More Discoveries
Explore Other Products
Browse additional items from our catalog
MBS6320605-01 0.2 mL
MGAT1, ID (MGAT1, GGNT1, GLCT1, GLYT1, MGAT, Alpha-1,3-mannosyl-glycoprotein 2-beta-N-acetylglucosaminyltransferase, N-glycosyl-oligosaccharide-glycoprotein N-acetylglucosaminyltransferase I) (FITC)
Sign In for Pricing
View Details