Improved therapeutic efficacy in two mouse models of methylmalonic acidemia (MMA) using a second-generation mRNA therapy
Kimberly A. Coughlan, Marianne Eybye, Nicholas Henderson, Christine DeAntonis, Andrea Frassetto, Erin Hanahoe, Tatiana Ketova, Eric Jacquinet, Vladimir Presnyak, Ruchi Jain, John Marshall, Paolo G.V. Martini
Topics & Concepts
Methylmalonic acidemiaMethylmalonic acidMedicineChemistryInternal medicineHomocysteineMetabolism and Genetic DisordersMitochondrial Function and PathologyPeroxisome Proliferator-Activated Receptors