Drisapersen sodium, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.
分子式
C211H256N76Na19O119P19S19
分子量
6978 (free acid)
CAS号
1181666-20-5
运输条件
Room temperature in continental US; may vary elsewhere.
储存方式
-20°C, sealed storage, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)