Биологическая активность
ML372 is a малая молекула SMN modulator that increases SMN protein in patient fibroblasts with EC50 of 37 nM; possesses good potency, pharmacokinetics, tolerance, and CNS penetration that are able to increase levels of SMN protein in several model клетка lines; increase SMN protein levels in vivo, restore motor function, and prolong survival of SMNΔ7 SMA Mice; improves the righting reflex and extended survival of a severe mouse model of SMA.
Оригинал на английском
ML372 is a small molecule SMN modulator that increases SMN protein in patient fibroblasts with EC50 of 37 nM; possesses good potency, pharmacokinetics, tolerance, and CNS penetration that are able to increase levels of SMN protein in several model cell lines; increase SMN protein levels in vivo, restore motor function, and prolong survival of SMNΔ7 SMA Mice; improves the righting reflex and extended survival of a severe mouse model of SMA.
Химические свойства
- CAS №
- 1331745-61-9
- Брутто-формула
- C₁₈H₂₀N₂O₄S
- Мол. масса
- 360.43 г/моль
- SMILES
- O=C(O)C(CC1)CCN1C2=NC=C(S2)C3=CC(OCCCO4)=C4C=C3
- Синонимы
- ML 372; ML 372; ML 372
3D-структура молекулы
Вращайте мышью · Колесо — масштаб
Хранение и физические свойства
- Условия хранения
- 2 года -20°C порошок, 2 недели 4°C в ДМСО, 6 мес. -80°C в ДМСО Publication: 1. Burnett BG, et al. Probe Reports from the NIH Molecular Libraries Program [Internet]. 2. Abera MB, et al. JCI Insight. 2016 Nov 17;1(19):e88427.
Безопасность и документация
Только для научно-исследовательского применения. Не предназначено для использования у пациентов или в качестве лекарственного средства.