Tissue-specific expression of an anti-proliferative hybrid transgene from the human smooth muscle alpha-actin promoter suppresses smooth muscle cell proliferation and neointima formation

Gene Ther. 2001 Dec;8(24):1847-54. doi: 10.1038/sj.gt.3301603.

Abstract

The retinoblastoma protein (Rb), a key regulator of cell cycle progression, can bind the transcription factor E2F converting it from a positive transcriptional factor capable of driving cells into S phase into a negative complex which arrests cells in G1. We have created a potent transcriptional repressor of E2F-dependent transcription by fusing the C-terminal fragment of Rb (p56) to the DNA and DP1-binding domains of E2F. Because the expression of E2F/56 fusion protein from a constitutive promoter was incompatible with virus growth, adenovirus constructs were prepared where transgenes were expressed from a fragment of the smooth muscle alpha-actin (SMA) promoter. Immunoblot and beta-galactosidase staining demonstrated smooth muscle-specific expression of this transcriptional element in vitro. The SMA-p56 and SMA-E2F/p56 adenoviral constructs also induced G0/G1 cell cycle arrest specifically in smooth muscle cells. Following administration to rat tissues, the SMA-beta-galactosidase construct exhibited expression in balloon-injured carotid arteries, but not in liver, bladder or skeletal muscle. Local delivery of the SMA-E2F/p56 adenoviral construct to balloon-injured carotid arteries inhibited intimal hyperplasia. Our results demonstrate that local delivery of the SMA-E2F/p56 adenoviral construct can limit intimal hyperplasia in balloon-injured vessels, while avoiding toxicity that could occur from the dissemination and expression of the viral transgene.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Actins / genetics*
  • Adenoviridae / genetics*
  • Angioplasty, Balloon / adverse effects
  • Animals
  • Artificial Gene Fusion / methods
  • Carotid Arteries / pathology
  • Cell Cycle
  • Cell Cycle Proteins*
  • DNA-Binding Proteins*
  • E2F Transcription Factors
  • Genetic Therapy / methods*
  • Genetic Vectors / administration & dosage*
  • Humans
  • Hyperplasia / prevention & control
  • Muscle, Smooth, Vascular / pathology*
  • Rats
  • Retinoblastoma Protein / genetics
  • Transcription Factors / genetics
  • Transgenes
  • Tunica Intima / pathology
  • beta-Galactosidase / genetics*

Substances

  • Actins
  • Cell Cycle Proteins
  • DNA-Binding Proteins
  • E2F Transcription Factors
  • Retinoblastoma Protein
  • Transcription Factors
  • beta-Galactosidase