Oligopeptide complex for targeted non-viral gene delivery to adipocytes

Nat Mater. 2014 Dec;13(12):1157-64. doi: 10.1038/nmat4092. Epub 2014 Oct 5.

Abstract

Commercial anti-obesity drugs acting in the gastrointestinal tract or the central nervous system have been shown to have limited efficacy and severe side effects. Anti-obesity drug development is thus focusing on targeting adipocytes that store excess fat. Here, we show that an adipocyte-targeting fusion-oligopeptide gene carrier consisting of an adipocyte-targeting sequence and 9-arginine (ATS-9R) selectively transfects mature adipocytes by binding to prohibitin. Injection of ATS-9R into obese mice confirmed specific binding of ATS-9R to fat vasculature, internalization and gene expression in adipocytes. We also constructed a short-hairpin RNA (shRNA) for silencing fatty-acid-binding protein 4 (shFABP4), a key lipid chaperone in fatty-acid uptake and lipid storage in adipocytes. Treatment of obese mice with ATS-9R/shFABP4 led to metabolic recovery and body-weight reduction (>20%). The ATS-9R/shFABP4 oligopeptide complex could prove to be a safe therapeutic approach to regress and treat obesity as well as obesity-induced metabolic syndromes.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adipocytes / drug effects*
  • Adipocytes / metabolism*
  • Animals
  • Fatty Acid-Binding Proteins / genetics*
  • Fatty Acid-Binding Proteins / metabolism
  • Fatty Acids / pharmacokinetics
  • Gene Expression
  • Gene Silencing
  • Gene Transfer Techniques*
  • Mice
  • Molecular Targeted Therapy / methods
  • Obesity / drug therapy*
  • Obesity / metabolism
  • Oligopeptides / administration & dosage*
  • Oligopeptides / pharmacokinetics
  • Prohibitins
  • RNA, Small Interfering / metabolism
  • Repressor Proteins / genetics*
  • Repressor Proteins / metabolism
  • Transfection / methods

Substances

  • FABP4 protein, human
  • Fatty Acid-Binding Proteins
  • Fatty Acids
  • Oligopeptides
  • Prohibitins
  • RNA, Small Interfering
  • Repressor Proteins