Retinal gene therapy: Current progress and future prospects

Cristy A. Ku, Mark E. Pennesi

Research output: Contribution to journalArticlepeer-review

13 Scopus citations


Clinical trials treating inherited retinal dystrophy caused by RPE65 mutations had put retinal gene therapy at the forefront of gene therapy. Both successes and limitations in these clinical trials have fueled developments in gene vectors, which continue to further advance the field. These novel gene vectors aim to more safely and efficiently transduce retinal cells, expand the gene packaging capacity of adeno-associated virus, and utilize new strategies to correct the varying mechanisms of dysfunction found with inherited retinal dystrophies. With recent clinical trials and numerous pre-clinical studies utilizing these novel vectors, the future of ocular gene therapy continues to hold vast potential.

Original languageEnglish (US)
Article number1035711
Pages (from-to)281-299
Number of pages19
JournalExpert Review of Ophthalmology
Issue number3
StatePublished - Jun 1 2015


  • RPE65
  • adeno-associated virus
  • equine infectious anemia virus
  • gene therapy
  • inherited retinal dystrophies

ASJC Scopus subject areas

  • Biomedical Engineering
  • Ophthalmology
  • Optometry


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