基因修改的VIII因子mRNA与银河系脂质纳米颗粒一起输送,作为A型血友病的蛋白质替代疗法

Porkizhi Arjunan1,2, Gokulnath Mahalingam1, Priyanka Sankar1

  • 1Centre for Stem Cell Research (CSCR) (a unit of inStem, Bengaluru), CMC Campus, Vellore, 632002, TN, India. srujankm@cmcvellore.ac.in.

Biomaterials science
|August 30, 2024
PubMed
概括

化学修饰的信使RNA (mRNA) 与N1-甲基伪尿素增强了对血友病A的蛋白质替代疗法. 这种方法提高了治疗效果,可能减少了频繁输注XIII因子的需要.