Triptolide treatment reduces Alzheimer's disease (AD)-like pathology through inhibition of BACE1 in a transgenic mouse model of AD

Dis Model Mech. 2014 Dec;7(12):1385-95. doi: 10.1242/dmm.018218.

Abstract

The complex pathogenesis of Alzheimer's disease (AD) involves multiple contributing factors, including amyloid β (Aβ) peptide accumulation, inflammation and oxidative stress. Effective therapeutic strategies for AD are still urgently needed. Triptolide is the major active compound extracted from Tripterygium wilfordii Hook.f., a traditional Chinese medicinal herb that is commonly used to treat inflammatory diseases. The 5-month-old 5XFAD mice, which carry five familial AD mutations in the β-amyloid precursor protein (APP) and presenilin-1 (PS1) genes, were treated with triptolide for 8 weeks. We observed enhanced spatial learning performances, and attenuated Aβ production and deposition in the brain. Triptolide also inhibited the processing of amyloidogenic APP, as well as the expression of βAPP-cleaving enzyme-1 (BACE1) both in vivo and in vitro. In addition, triptolide exerted anti-inflammatory and anti-oxidative effects on the transgenic mouse brain. Triptolide therefore confers protection against the effects of AD in our mouse model and is emerging as a promising therapeutic candidate drug for AD.

Keywords: 5XFAD mice; Alzheimer’s disease; Amyloid β; BACE1; Inflammation; Triptolide.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alzheimer Disease / drug therapy*
  • Amyloid Precursor Protein Secretases / genetics*
  • Amyloid beta-Protein Precursor / genetics
  • Animals
  • Anti-Inflammatory Agents / chemistry
  • Antioxidants / chemistry
  • Aspartic Acid Endopeptidases / genetics*
  • Brain / drug effects
  • Brain / metabolism
  • Disease Models, Animal
  • Diterpenes / pharmacology*
  • Epoxy Compounds / pharmacology
  • Female
  • Humans
  • Immunosuppressive Agents / pharmacology
  • Learning / drug effects
  • Maze Learning
  • Memory / drug effects
  • Mice
  • Mice, Inbred C57BL
  • Mice, Transgenic
  • Mutation
  • Phenanthrenes / pharmacology*
  • Plant Extracts / pharmacology

Substances

  • Amyloid beta-Protein Precursor
  • Anti-Inflammatory Agents
  • Antioxidants
  • Diterpenes
  • Epoxy Compounds
  • Immunosuppressive Agents
  • Phenanthrenes
  • Plant Extracts
  • triptolide
  • Amyloid Precursor Protein Secretases
  • Aspartic Acid Endopeptidases
  • BACE1 protein, human
  • Bace1 protein, mouse