Phase 1 Trials of PNPLA3 siRNA in I148M Homozygous Patients with MAFLD

N Engl J Med. 2024 Aug 1;391(5):475-476. doi: 10.1056/NEJMc2402341.
No abstract available

Publication types

  • Clinical Trial, Phase I
  • Letter
  • Randomized Controlled Trial

MeSH terms

  • Acyltransferases / antagonists & inhibitors
  • Acyltransferases / genetics
  • Adult
  • Aged
  • Female
  • Homozygote*
  • Humans
  • Liver / drug effects
  • Liver / pathology
  • Male
  • Middle Aged
  • Mutation
  • Non-alcoholic Fatty Liver Disease* / drug therapy
  • Non-alcoholic Fatty Liver Disease* / genetics
  • Non-alcoholic Fatty Liver Disease* / metabolism
  • Non-alcoholic Fatty Liver Disease* / pathology
  • Phospholipases A2, Calcium-Independent / antagonists & inhibitors
  • Phospholipases A2, Calcium-Independent / genetics
  • Polymorphism, Single Nucleotide
  • Precision Medicine / methods
  • Proof of Concept Study
  • RNA, Small Interfering* / administration & dosage
  • RNA, Small Interfering* / adverse effects
  • RNA, Small Interfering* / pharmacokinetics
  • Young Adult

Substances

  • Acyltransferases
  • Phospholipases A2, Calcium-Independent
  • PNPLA3 protein, human
  • RNA, Small Interfering