Gene therapy for diseases of the cornea - a review

Keryn A. Williams, Douglas J. Coster

Research output: Contribution to journalReview articlepeer-review

31 Citations (Scopus)


The cornea is particularly suited to gene therapy. The cornea is readily accessible, normally transparent, and is somewhat sequestrated from the general circulation and the systemic immune system. The principle of genetic therapy for the cornea is to use an appropriate vector system to transfer a gene to the cornea itself, or to the ocular environs, or systemically, so that a transgenic protein will be expressed that will modulate congenital or acquired disease. The protein may be structural such as a collagen, or functionally active such as an enzyme, cytokine or growth factor that may modulate a pathological process. Alternatively, gene expression may be silenced by the use of modalities such as antisense oligonucleotides. Interestingly, despite a very considerable amount of work in animal models, clinical translation directed to gene therapy of the human cornea has been minimal. This is in contrast to gene therapy for monogenic inherited diseases of the retina, where promising early results of clinical trials for Leber's congenital amaurosis have already been published and a number of other trials are ongoing.

Original languageEnglish
Pages (from-to)93-103
Number of pages11
JournalClinical and Experimental Ophthalmology
Issue number2
Publication statusPublished or Issued - Mar 2010
Externally publishedYes


  • Cornea
  • Gene transfer
  • Regulatory issue
  • Safety
  • Transgene delivery
  • Transgenic protein

ASJC Scopus subject areas

  • Ophthalmology

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