Corneal gene therapy

Eytan A. Klausner, Dan Peer, Robert L. Chapman, Richard F. Multack, Shridhar V. Andurkar

Research output: Contribution to journalReview article

63 Scopus citations

Abstract

Gene therapy to the cornea can potentially correct inherited and acquired diseases of the cornea. Factors that facilitate corneal gene delivery are the accessibility and transparency of the cornea, its stability ex vivo and the immune privilege of the eye. Initial corneal gene delivery studies characterized the relationship between intraocular modes of administration and location of reporter gene expression. The challenge of achieving effective topical gene transfer, presumably due to tear flow, blinking and low penetration of the vector through epithlelial tight junctions left no alternative but invasive administration to the anterior chamber and corneal stroma. DNA vaccination, RNA interference and gene transfer of cytokines, growth factors and enzymes modulated the corneal microenvironment. Positive results were obtained in preclinical studies for prevention and treatment of corneal graft rejection, neovascularization, haze and herpetic stromal keratitis. These studies, corneal gene delivery systems and modes of administration, and considerations regarding the choice of animal species used are the focus of this review. Opportunities in the field of corneal gene therapy lie in expanding the array of corneal diseases investigated and in the implementation of recent designs of safer vectors with reduced immunogenicity and longer duration of gene expression.

Original languageEnglish (US)
Pages (from-to)107-133
Number of pages27
JournalJournal of Controlled Release
Volume124
Issue number3
DOIs
StatePublished - Dec 20 2007

Keywords

  • Cornea
  • Corneal graft rejection
  • Corneal neovascularization
  • Eye
  • Gene therapy
  • Herpetic stromal keratitis

ASJC Scopus subject areas

  • Pharmaceutical Science

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