Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency
间歇性脂质纳米颗粒 mRNA 给药可预防与精氨酸酶缺乏相关的皮质髓鞘形成障碍
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.5
doi:10.1016/j.omtn.2022.04.012
Suhail Khoja, Xiao-Bo Liu, Brian Truong, Matthew Nitzahn, Jenna Lambert, Adam Eliav, Eram Nasser, Emma Randolph, Kristine E Burke, Rebecca White, Xuling Zhu, Paolo G V Martini, Itzhak Nissim, Stephen D Cederbaum, Gerald S Lipshutz
