Branaplam (LMI070) (Synonyms: Branaplam, NVS-SM1) |
| Catalog No.GC32702 |
Branaplam(LMI070)(LMI070, NVS-SM1)은 SMN에 대해 EC50이 20nM인 매우 강력하고 선택적이고 경구 활성인 생존 운동 뉴런-2(SMN2) 스플라이싱 조절제입니다. Branaplam(LMI070)은 IC50이 6.3 μ인 hERG(human-ether-a-go-go-related gene)를 억제합니다. Branaplam(LMI070)은 전체 길이의 SMN 단백질을 증가시키고 중증 척수성 근위축증(SMA) 마우스 모델에서 생존을 연장합니다.
Products are for research use only. Not for human use. We do not sell to patients.
Cas No.: 1562338-42-4
Sample solution is provided at 25 µL, 10mM.
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Branaplam (LMI070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator.
[1]. Palacino J, et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat Chem Biol. 2015 Jul;11(7):511-517.
Average Rating: 5 (Based on Reviews and 11 reference(s) in Google Scholar.)
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