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

Hum Gene Ther. 2024 Sep;35(17-18):726-733. doi: 10.1089/hum.2024.047. Epub 2024 Aug 5.
No abstract available

Keywords: BCKDH; branched-chain amino acids; gene therapy; lipid nanoparticle; maple syrup urine disease; messenger RNA.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acids, Branched-Chain* / blood
  • Animals
  • Disease Models, Animal*
  • Genetic Therapy / methods
  • Humans
  • Lipids / blood
  • Liposomes
  • Maple Syrup Urine Disease* / genetics
  • Maple Syrup Urine Disease* / therapy
  • Mice
  • Nanoparticles*
  • RNA, Messenger* / genetics
  • RNA, Messenger* / metabolism

Substances

  • Amino Acids, Branched-Chain
  • RNA, Messenger
  • Lipid Nanoparticles
  • Lipids
  • Liposomes