Koeberl D, Schulze A, Sondheimer N, Lipshutz GS, Geberhiwot T, Li L et al.
Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia.
Nature. 2024 ;628:872-877
An D, Frassetto A, Jacquinet E, Eybye M, Milano J, DeAntonis C et al.
Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia.
EBioMedicine. 2019 Jul;45:519-528

