Retinal gene therapy

Current progress and future prospects

Cristy A. Ku, Mark Pennesi

Research output: Contribution to journalArticle

10 Citations (Scopus)

Abstract

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
Volume10
Issue number3
DOIs
StatePublished - Jun 1 2015

Fingerprint

Gene therapy
Retinal Dystrophies
Genetic Therapy
Genes
Clinical Trials
Dependovirus
Product Packaging
Viruses
Packaging
Cells
Mutation

Keywords

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

ASJC Scopus subject areas

  • Ophthalmology
  • Biomedical Engineering
  • Optometry

Cite this

Retinal gene therapy : Current progress and future prospects. / Ku, Cristy A.; Pennesi, Mark.

In: Expert Review of Ophthalmology, Vol. 10, No. 3, 1035711, 01.06.2015, p. 281-299.

Research output: Contribution to journalArticle

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