Neuroprotection using gene therapy to induce vascular endothelial growth factor-A expression

Gene Ther. 2009 Nov;16(11):1292-9. doi: 10.1038/gt.2009.111. Epub 2009 Sep 3.

Abstract

Engineered zinc-finger protein (ZFP) transcription factors induce the expression of endogenous genes and can be remotely delivered using adenoviral vectors. One such factor, Ad-32Ep65-Flag (Ad-p65), targets and induces expression of vascular endothelial growth factor (VEGF; also called VEGF-A) splice variants in their normal biological stoichiometry. We show that Ad-p65 transfection of primary motor neurons results in VEGF variant expression and a significant increase in axon outgrowth in these cells. Given the neuroprotective effects of VEGF and its ability to increase neurite outgrowth, we examined the efficacy of Ad-p65 to enhance motor neuron regeneration in vivo using rats that have undergone recurrent laryngeal nerve (RLN)-crush injury. Injection of Ad-p65 after RLN crush accelerated the return of vocal fold mobility and the percentage of nerve-endplate contacts in the thyroarytenoid muscle. Overall, adenoviral delivery of an engineered ZFP transcription factor inducing VEGF-A splice variant expression enhances nerve regeneration. ZFP transcription factor gene therapy to increase expression of the full complement of VEGF-A splice variants is a promising avenue for the treatment of nerve injury and neurodegeneration.

Publication types

  • Evaluation Study
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenoviridae / genetics
  • Animals
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Motor Endplate / physiology
  • Motor Neurons / metabolism
  • Motor Neurons / physiology*
  • Nerve Crush
  • Nerve Regeneration / genetics
  • Rats
  • Rats, Sprague-Dawley
  • Recurrent Laryngeal Nerve Injuries
  • Transfection
  • Up-Regulation
  • Vascular Endothelial Growth Factor A / genetics*
  • Vascular Endothelial Growth Factor A / metabolism
  • Vocal Cord Paralysis / metabolism
  • Vocal Cord Paralysis / physiopathology
  • Vocal Cord Paralysis / therapy*
  • Vocal Cords / physiology
  • Zinc Fingers / genetics

Substances

  • VEGFA protein, human
  • Vascular Endothelial Growth Factor A