abstract |
The present disclosure relates to a polynucleotide comprising an open reading frame of a linked nucleoside encoding a human methylmalonyl CoA mutase precursor, a human methylmalonyl CoA mutase (MCM) mature form or a functional fragment thereof. In some embodiments, the disclosure includes a method of treating methylmalonic acidemia in a subject in need thereof, comprising administering mRNA encoding an MCM polypeptide. |