Биологическая активность
L2 is a redox-responsive липид engineered for ultra-мощный siRNA delivery. With shorter hexyl (C6) tails and a carbamate linker, it demonstrates the fastest biodegradation (liver half-life: 2.6 days) among tested lipids. L2 achieves >80% FVII gene knockdown at just 0.01 mg/kg—surpassing both L1 and MC3 in siRNA potency. Its higher apparent pKa (6.90) enhances endosomal disruption, correlating with strong in vitro hEPO expression. While slightly less effective for mRNA than L1, L2’s unmatched siRNA silencing efficiency, rapid cytosolic self-immolation, and low cytotoxicity position it as a leading candidate for RNAi therapeutics targeting hepatic diseases.
Оригинал на английском
L2 is a redox-responsive lipid engineered for ultra-potent siRNA delivery. With shorter hexyl (C6) tails and a carbamate linker, it demonstrates the fastest biodegradation (liver half-life: 2.6 days) among tested lipids. L2 achieves >80% FVII gene knockdown at just 0.01 mg/kg—surpassing both L1 and MC3 in siRNA potency. Its higher apparent pKa (6.90) enhances endosomal disruption, correlating with strong in vitro hEPO expression. While slightly less effective for mRNA than L1, L2’s unmatched siRNA silencing efficiency, rapid cytosolic self-immolation, and low cytotoxicity position it as a leading candidate for RNAi therapeutics targeting hepatic diseases.
Химические свойства
- Брутто-формула
- C₄₂H₈₂O₆N₂S₂
- Мол. масса
- 775.25 г/моль
- SMILES
- CCCCCCC(CCCCCC)OC(=O)CCCN(CCCC(=O)OC(CCCCCC)CCCCCC)C(=O)OCCSSCCCN(C)C
- Синонимы
- ATX-L2， ATXL2
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: Design and Evaluation of Biodegradable Self-Immolative Lipids for RNA Delivery-R Mukthavaram, A Sagi, H Hong, R Recatto… - Advanced healthcare materials
Безопасность и документация
Только для научно-исследовательского применения. Не предназначено для использования у пациентов или в качестве лекарственного средства.