Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine model

Xuejun Zhao, Al‐Walid Mohsen, Stephanie J. Mihalik, Keaton J. Solo, Ermal Aliu, Huifang Shi, Shakuntala Basu, Catherine Kochersperger, Clinton Van’t Land, Anuradha Karunanidhi, Kimberly A. Coughlan, Summar Siddiqui, Lisa Rice, Shawn Hillier, Eleonora Guadagnin, Paloma H. Giangrande, Paolo G.V. Martini, Jerry Vockley