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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Antiangiogenic Eye Gene Therapy.

Thomas J Corydon1

  • 1Department of Biomedicine, Aarhus University , Aarhus C, Denmark .

Human Gene Therapy
|July 30, 2015
PubMed
Summary

Ocular gene therapy shows promise for treating eye diseases like inherited retinal dystrophies and age-related macular degeneration. Advances in gene delivery vectors and the eye's unique properties facilitate its clinical translation.

Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Gene therapy, using genetic material to treat disease, has evolved over two decades.
  • The eye's accessibility, compartmentalization, and immune privilege make it an ideal target for gene therapy.
  • Significant progress in retinal cell transduction using recombinant adeno-associated virus (AAV) and lentivirus vectors has been achieved.

Purpose of the Study:

  • To review the progress of ocular gene therapy.
  • To emphasize the potential of RNA interference (RNAi) and protein-based antiangiogenic gene therapy.
  • To explore applications in treating exudative age-related macular degeneration.

Main Methods:

  • Review of existing literature and clinical trial data on ocular gene therapy.

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  • Focus on gene supplementation and silencing strategies.
  • Examination of nonintegrating viral vectors, including AAV and lentivirus.

    • Main Results:

    • Gene therapy has demonstrated efficacy in treating genetic conditions.
    • Ocular gene therapy has shown significant advancements, particularly in gene supplementation and silencing.
    • In vivo studies suggest safety and effectiveness for inherited retinal dystrophies, ocular angiogenesis, and inflammation.

    Conclusions:

    • Ocular gene therapy is a rapidly advancing field with strong clinical potential.
    • The unique characteristics of the eye facilitate gene therapy applications.
    • Antiangiogenic gene therapy holds promise for treating exudative age-related macular degeneration.