Name: Golodirsen
Brand: TargetMol
SKU: T38860
Rating: 4.5 (1 reviews)

规格

价格

库存

数量

1 mg

¥ 3,130

2-4周

5 mg

¥ 7,830

2-4周

生物活性

产品描述	Golodirsen (SRP-4053) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) class that restores the dystrophin gene reading frame in Duchenne muscular dystrophy by modulating pre-mRNA splicing to induce skipping of exon 53, thereby enabling partial dystrophin expression and restoring anti-myostatin protein levels, which supports its application as a molecular tool for studying exon-skipping strategies and disease-modifying approaches in DMD research.
体外活性	Golodirsen specifically targets exon 53 of dystrophin pre-mRNA, resulting in its exclusion from the final mRNA product. Skipping of exon 53 results in restoration of the mRNA reading frame and leads to the production of a partially internally deleted dystrophin protein with intact C and N-terminal regions[2].
别名	戈洛地生, SRP-4053, SRP4053

化学信息

CAS No.	1422959-91-8
Sequence	RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(G-m5U-m5U-G-C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-GG-m5U-G-m5U-m5U-C), 5'-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,Ndimethylphosphonamidate]

储存&溶解度

存储	keep away from moisture \| In solvent: -80°C for 1 year \| Shipping with blue ice/Shipping at ambient temperature.
溶解度信息	H2O: 80 mg/mL, Sonication is recommended.

Golodirsen