For research use only. Not for therapeutic Use.
Ulefnersen（Cat No.:I043362）is an investigational antisense oligonucleotide designed to target and skip exon 51 of the dystrophin gene, offering a potential treatment for Duchenne muscular dystrophy (DMD) in patients with specific genetic mutations. By promoting exon skipping, ulefnersen aims to restore the production of a shortened but functional form of dystrophin, a protein essential for muscle function. Clinical studies have shown that ulefnersen can increase dystrophin expression in muscle tissue, offering hope for slowing the progression of DMD. It is being evaluated in clinical trials for safety, efficacy, and long-term benefits.
| CAS Number | 2589926-25-8 |
| Molecular Formula | C230H321N68O126P19S13 |
| Purity | ≥95% |
| Chemistry Calculators | Dilution Calculator In vivo Formulation Calculator Molarity Calculator Molecular Weight Calculator Reconstitution Calculator |
