Lipid Nanoparticle mRNA Therapy Improves Survival and Reduces Serum Branched-Chain Amino Acids in Mouse Models of Maple Syrup Urine Disease.

Jenny A GreigMatthew JennisAditya DandekarJoanna K ChorazeczewskiNesteene ParamMeardey SoMohamad NayalPeter BellKimberly CoughlanMinjung ChoiPaloma H GiangrandePaolo G V MartiniJames M Wilson

Published in: Human gene therapy (2024)

Keyphrases

amino acid
mouse model
binding protein
free survival
iron oxide
mesenchymal stem cells
bone marrow