Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine model
合成mRNA可挽救患者成纤维细胞和鼠模型中的极长链酰基辅酶A脱氢酶缺乏症。
期刊:Molecular Genetics and Metabolism
影响因子:3.5
doi:10.1016/j.ymgme.2022.106982
Zhao, Xue-Jun; Mohsen, Ai-Walid; Mihalik, Stephanie; Solo, Keaton; Aliu, Ermal; Shi, Huifang; Basu, Shakuntala; Kochersperger, Catherine; Van't Land, Clinton; Karunanidhi, Anuradha; Coughlan, Kimberly A; Siddiqui, Summar; Rice, Lisa M; Hillier, Shawn; Guadagnin, Eleonora; Giangrande, Paloma H; Martini, Paolo G V; Vockley, Jerry
