Systemic mRNA-LNP administration in fetuses improves survival in a mouse model of spinal muscular atrophy.
在脊髓性肌萎缩症小鼠模型中，对胎儿进行全身性 mRNA-LNP 给药可提高存活率。
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2025.101599
Carpenter Marco D, Kus Nicole, Choi Rosa B, Said Hooda, Dumitru Ana Maria, Bauer Jackson H, Wang Samantha, Luks Valerie, Teerdhala Sriya, Shah Aarsha, Zoltick Philip W, Alameh Mohamad G, Peranteau William
