BioCentury
ARTICLE | Distillery Therapeutics

Endocrine / Metabolic

January 9, 2018 10:04 PM UTC

Mouse studies suggest a lipid nanoparticle (LNP)-encapsulated mRNA encoding MUT (mRNA-3704) could help treat methylmalonic acidemia (MMA), which is caused by MUT deficiency. In a mouse model of MMA, a...