Биологическая активность
A33-D268 is an ионизируемый липид derived from a 2-aminoimidazole (AM) core, featuring an asymmetric hydrophobic tail structure designed to optimize mRNA interactions. Selected as the top candidate from a 21-липид library through molecular dynamics (MD) simulations, it demonstrated superior performance: low root mean square deviation (RMSD) and the smallest radius of gyration (Rg), indicating tight mRNA compaction, alongside moderate electrostatic energy and above-average hydrogen bonding for stable encapsulation. Formulated into LNPs, it achieves efficient muscle-specific transfection post-intramuscular injection, rivaling commercial ALC-0315 LNPs. Crucially, it exhibits 29-fold lower hepatic off-target expression, attributed to its inability to leverage serum protein coronas for liver tropism.
Оригинал на английском
A33-D268 is an ionizable lipid derived from a 2-aminoimidazole (AM) core, featuring an asymmetric hydrophobic tail structure designed to optimize mRNA interactions. Selected as the top candidate from a 21-lipid library through molecular dynamics (MD) simulations, it demonstrated superior performance: low root mean square deviation (RMSD) and the smallest radius of gyration (Rg), indicating tight mRNA compaction, alongside moderate electrostatic energy and above-average hydrogen bonding for stable encapsulation. Formulated into LNPs, it achieves efficient muscle-specific transfection post-intramuscular injection, rivaling commercial ALC-0315 LNPs. Crucially, it exhibits 29-fold lower hepatic off-target expression, attributed to its inability to leverage serum protein coronas for liver tropism.
Химические свойства
- Брутто-формула
- C₄₂H₈₀O₄N₄
- Мол. масса
- 705.13 г/моль
- SMILES
- CCCCCCCCC(CCCCCC)C(=O)OCCCN(CCCOC(=O)C(CCCCCC)CCCCCCCC)CC1=CNC(N)=N1
- Синонимы
- Lipid A33 D268
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
Безопасность и документация
Только для научно-исследовательского применения. Не предназначено для использования у пациентов или в качестве лекарственного средства.