AAV2/8-humanFOXP3 gene therapy shows robust anti-atherosclerosis efficacy in LDLR-KO mice on high cholesterol diet

J Transl Med. 2015 Jul 18:13:235. doi: 10.1186/s12967-015-0597-7.

Abstract

Inflammation is a key etiologic component in atherogenesis. Previously we demonstrated that adeno-associated virus (AAV) 2/8 gene delivery of Netrin1 inhibited atherosclerosis in the low density lipoprotein receptor knockout mice on high-cholesterol diet (LDLR-KO/HCD). One important finding from this study was that FOXP3 was strongly up-regulated in these Netrin1-treated animals, as FOXP3 is an anti-inflammatory gene, being the master transcription factor of regulatory T cells. These results suggested that the FOXP3 gene might potentially be used, itself, as an agent to limit atherosclerosis. To test this hypothesis AAV2/8 (AAV)/hFOXP3 or AAV/Neo (control) gene therapy virus were tail vein injected into the LDLR-KO/HCD animal model. It was found that hFOXP3 gene delivery was associated with significantly lower HCD-induced atherogenesis, as measured by larger aortic lumen cross sectional area, thinner aortic wall thickness, and lower aortic systolic blood velocity compared with Neo gene-HCD-treated controls. Moreover these measurements taken from the hFOXP3/HCD-treated animals very closely matched those measurements taken from the normal diet (ND) control animals. These data strongly suggest that AAV/hFOXP3 delivery gave a robust anti-atherosclerosis therapeutic effect and further suggest that FOXP3 be examined more stringently as a therapeutic gene for clinical use.

Publication types

  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Animals
  • Aorta / diagnostic imaging
  • Aorta / pathology
  • Atherosclerosis / blood
  • Atherosclerosis / physiopathology
  • Atherosclerosis / therapy*
  • Cholesterol, Dietary / blood*
  • Dependovirus*
  • Diet
  • Forkhead Transcription Factors / genetics*
  • Genetic Therapy / methods
  • Humans
  • Inflammation / pathology
  • Male
  • Mice
  • Mice, Knockout
  • Receptors, LDL / genetics*
  • T-Lymphocytes, Regulatory
  • Transgenes
  • Ultrasonography

Substances

  • Cholesterol, Dietary
  • FOXP3 protein, human
  • Forkhead Transcription Factors
  • Receptors, LDL