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Gene therapy for ocular angiogenesis.

James W B Bainbridge1, Ajay R Mistry, Adrian J Thrasher

  • 1Department of Molecular Genetics, Institute of Ophthalmology, University College London, 11-43 Bath Street, London EC1V 9EL, U.K. j.bainbridge@ucl.ac.uk

Clinical Science (London, England : 1979)
|February 7, 2003
PubMed
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Gene therapy using adeno-associated virus vectors offers a promising approach for treating ocular neovascularization, a leading cause of blindness. This method allows for sustained, localized delivery of therapeutic proteins to the retina.

Area of Science:

  • Ophthalmology
  • Molecular Biology
  • Gene Therapy

Background:

  • Ocular neovascularization, common in diabetic retinopathy and age-related macular degeneration, causes significant vision loss.
  • Current treatments for these conditions have limited efficacy and notable adverse effects.
  • Angiostatic molecules show therapeutic potential but face challenges with systemic administration and short intraocular effect duration.

Purpose of the Study:

  • To evaluate the potential of local gene transfer for sustained and regulated delivery of angiostatic proteins to the retina.
  • To investigate the efficacy of recombinant adeno-associated virus (AAV)-mediated gene transfer for controlling ocular neovascularization.
  • To explore the use of hypoxia-responsive promoters for regulating therapeutic gene expression in the context of ocular neovascular disorders.

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Main Methods:

  • Utilized recombinant adeno-associated virus (AAV) vectors for local gene transfer in rodent models of ocular neovascularization.
  • Administered AAV vectors intraocularly to deliver genes encoding vascular endothelial growth factor inhibitors.
  • Conducted preclinical studies in a large animal model with subretinal delivery of AAV vectors to assess sustained gene expression.

Main Results:

  • Demonstrated that intraocular delivery of recombinant viruses carrying angiostatic protein genes can control retinal and choroidal neovascularization in rodent models.
  • Showed that AAV-mediated gene transfer of a vascular endothelial growth factor inhibitor effectively manages ocular neovascularization.
  • Confirmed efficient and sustained reporter gene expression in the outer retina following subretinal AAV vector delivery in a large animal model.

Conclusions:

  • Local gene transfer, particularly using AAV vectors, presents a viable strategy for sustained and targeted delivery of therapeutic agents for ocular neovascular diseases.
  • Hypoxia-responsive promoters offer a method for regulating gene expression, potentially minimizing adverse effects in clinical applications.
  • Preclinical successes pave the way for clinical trials of gene therapy in managing vision-threatening ocular neovascular disorders.