Gene Therapy and Gene Editing for the Corneal Dystrophies

Keryn A. Williams, Yazad Irani

Research output: Contribution to journalReview article

5 Citations (Scopus)

Abstract

Despite ever-increasing understanding of the genetic underpinnings of many corneal dystrophies, gene therapy designed to ameliorate disease has not yet been reported in any human patient. In this review, we explore the likely reasons for this apparent failure of translation. We identify the requirements for success: the genetic defect involved must have been identified and mapped, vision in the affected patient must be significantly impaired or likely to be impaired, no better or equivalently effective treatment must be available, the treatment must be capable of modulating corneal pathology, and delivery of the construct to the appropriate cell must be practicable. We consider which of the corneal dystrophies might be amenable to treatment by genetic manipulations, summarize existing therapeutic options for treatment, and explore gene editing using clustered regularly interspaced short palindromic repeat/Cas and other similar transformative technologies as the way of the future. We then summarize recent laboratory-based advances in gene delivery and the development of in vitro and in vivo models of the corneal dystrophies. Finally, we review recent experimental work that has increased our knowledge of the pathobiology of these conditions.

LanguageEnglish
Pages312-316
Number of pages5
JournalAsia-Pacific Journal of Ophthalmology
Volume5
Issue number4
DOIs
Publication statusPublished - 1 Jul 2016
Externally publishedYes

Keywords

  • CRISPR/Cas
  • corneal dystrophy
  • gene editing
  • gene therapy
  • transformative technology

ASJC Scopus subject areas

  • Ophthalmology

Cite this

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Gene Therapy and Gene Editing for the Corneal Dystrophies. / Williams, Keryn A.; Irani, Yazad.

In: Asia-Pacific Journal of Ophthalmology, Vol. 5, No. 4, 01.07.2016, p. 312-316.

Research output: Contribution to journalReview article

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