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Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and Researchers

Geoffrey A Casey1, Kimberly M Papp2, Ian M MacDonald1,3

  • 1Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Canada.

Journal of Ophthalmic & Vision Research
|September 1, 2020
PubMed

Insights

Ocular gene therapy offers hope for inherited eye diseases. While challenges like immune responses persist, ongoing research and clinical trials are paving the way for effective treatments.

Area of Science:

  • Ophthalmology
  • Genetics
  • Immunology

Background:

  • Heritable ocular disorders represent a significant unmet medical need.
  • Advances in gene therapy have enabled clinical trials for previously untreatable conditions.
  • Leber congenital amaurosis treated with LUXTURNA serves as a pioneering example.

Purpose of the Study:

  • To review the current landscape of ocular gene therapy.
  • To discuss patient perspectives, therapeutic strategies, and vector delivery systems.
  • To address the persistent challenge of immune responses limiting treatment efficacy.

Main Methods:

  • Review of pre-clinical studies, including biochemical, cellular, and animal models.
  • Analysis of clinical trial data and patient outcomes.
  • Discussion of gene replacement and gene editing strategies.
  • Evaluation of adeno-associated virus vectors and ocular delivery routes.

Main Results:

  • Clinical trials are emerging for various heritable ocular disorders.
  • Immune reactions to vectors remain a key limitation in achieving optimal efficacy.
  • The success of LUXTURNA highlights the potential of ocular gene therapy.

Conclusions:

  • Ocular gene therapy is progressing towards widespread clinical use for inherited eye diseases.
  • Further research into immune mechanisms and novel vector design is crucial.
  • Managing patient expectations regarding current limitations is essential for successful implementation.

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