Биологическая активность
Di-Ellipticine-RIBOTAC TFA (Compound 7) is a RNA RIBOTAC degrader, capable of specifically связывание and degrading r(G4C2)exp. Di-Ellipticine-RIBOTAC TFA only cuts the expanded GGGGCC repeat RNA in the pathogenic factor of amyotrophic lateral sclerosis/ frontotemporal dementia (c9ALS/FTD) - the chromosome 9 open reading frame 72 (9orf72) gene (r(G4C2)exp), and has no effect on normal short repetitive sequences. Di-Ellipticine-RIBOTAC TFA significantly improves the pathological phenotype of c9ALS/FTD in cells and mouse models. Di-Ellipticine-RIBOTAC TFA can be used for the study of c9ALS/FTD.
Оригинал на английском
Di-Ellipticine-RIBOTAC TFA (Compound 7) is a RNA RIBOTAC degrader, capable of specifically binding and degrading r(G4C2)exp. Di-Ellipticine-RIBOTAC TFA only cuts the expanded GGGGCC repeat RNA in the pathogenic factor of amyotrophic lateral sclerosis/ frontotemporal dementia (c9ALS/FTD) - the chromosome 9 open reading frame 72 (9orf72) gene (r(G4C2)exp), and has no effect on normal short repetitive sequences. Di-Ellipticine-RIBOTAC TFA significantly improves the pathological phenotype of c9ALS/FTD in cells and mouse models. Di-Ellipticine-RIBOTAC TFA can be used for the study of c9ALS/FTD.
Химические свойства
- CAS №
- 2767983-77-5
- Брутто-формула
- C₈₀H₈₈F₃N₇O₁₈S
- Мол. масса
- 1524.65 г/моль
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.