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Liu MM, Tuo J, Chan CC. Republished review: Gene therapy for ocular diseases. Postgrad Med J. 2011;87(1029):487-95doi: 10.1136/pgmj.2009.174912rep.
Liu, M. M., Tuo, J., & Chan, C. C. (2011). Republished review: Gene therapy for ocular diseases. Postgraduate medical journal, 87(1029), 487-95. https://doi.org/10.1136/pgmj.2009.174912rep
Liu, Melissa M, et al. "Republished review: Gene therapy for ocular diseases." Postgraduate medical journal vol. 87,1029 (2011): 487-95. doi: https://doi.org/10.1136/pgmj.2009.174912rep
Liu MM, Tuo J, Chan CC. Republished review: Gene therapy for ocular diseases. Postgrad Med J. 2011 Jul;87(1029):487-95. doi: 10.1136/pgmj.2009.174912rep. PMID: 21705775; PMCID: PMC3172160.
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Postgrad Med J. 2011 Jul;87(1029):487-95. doi: 10.1136/pgmj.2009.174912rep.

Republished review: Gene therapy for ocular diseases.

Postgraduate medical journal

Melissa M Liu, Jingsheng Tuo, Chi-Chao Chan

Affiliations

  1. Immunopathology Section, Laboratory of Immunology, National Eye Institute, National Institutes of Health, 10 Center Drive, Bldg 10, Rm 10N103, NIH/NEI, Bethesda, MD 20895-1857, USA.

PMID: 21705775 PMCID: PMC3172160 DOI: 10.1136/pgmj.2009.174912rep

Abstract

The eye is an easily accessible, highly compartmentalised and immune-privileged organ that offers unique advantages as a gene therapy target. Significant advancements have been made in understanding the genetic pathogenesis of ocular diseases, and gene replacement and gene silencing have been implicated as potentially efficacious therapies. Recent improvements have been made in the safety and specificity of vector-based ocular gene transfer methods. Proof-of-concept for vector-based gene therapies has also been established in several experimental models of human ocular diseases. After nearly two decades of ocular gene therapy research, preliminary successes are now being reported in phase 1 clinical trials for the treatment of Leber congenital amaurosis. This review describes current developments and future prospects for ocular gene therapy. Novel methods are being developed to enhance the performance and regulation of recombinant adeno-associated virus- and lentivirus-mediated ocular gene transfer. Gene therapy prospects have advanced for a variety of retinal disorders, including retinitis pigmentosa, retinoschisis, Stargardt disease and age-related macular degeneration. Advances have also been made using experimental models for non-retinal diseases, such as uveitis and glaucoma. These methodological advancements are critical for the implementation of additional gene-based therapies for human ocular diseases in the near future.

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