Optogenetic approaches to vision restoration

M P Simunovic; W Shen; J Y Lin; D A Protti; L Lisowski; M C Gillies

doi:10.1016/j.exer.2018.09.003

Optogenetic approaches to vision restoration

Exp Eye Res. 2019 Jan:178:15-26. doi: 10.1016/j.exer.2018.09.003. Epub 2018 Sep 13.

Authors

M P Simunovic¹, W Shen², J Y Lin³, D A Protti⁴, L Lisowski⁵, M C Gillies⁶

Affiliations

¹ Save Sight Institute, University of Sydney, 8 Macquarie St., Sydney, NSW, 2000, Australia; Retinal Unit, Sydney Eye Hospital, 8 Macquarie St., Sydney, NSW, 2000, Australia. Electronic address: matthew.simunovic@sydney.edu.au.
² Retinal Unit, Sydney Eye Hospital, 8 Macquarie St., Sydney, NSW, 2000, Australia.
³ Faculty of Health, University of Tasmania, Private Bag 23, Hobart, TAS, 7000, Australia.
⁴ Discipline of Physiology, University of Sydney, NSW, 2006, Australia.
⁵ Children's Medical Research Institute, University of Sydney, NSW, 2006, Australia; Military Institute of Hygiene and Epidemiology, 24-100, Puławy, Poland.
⁶ Save Sight Institute, University of Sydney, 8 Macquarie St., Sydney, NSW, 2000, Australia; Retinal Unit, Sydney Eye Hospital, 8 Macquarie St., Sydney, NSW, 2000, Australia.

PMID: 30218651
DOI: 10.1016/j.exer.2018.09.003

Abstract

Inherited retinal disease (IRD) affects about 1 in 3000 to 1 in 5000 individuals and is now believed to be the most common cause of blindness registration in developed countries. Until recently, the management of such conditions had been exclusively supportive. However, advances in molecular biology and medical engineering have now seen the rise of a variety of approaches to restore vision in patients with IRDs. Optogenetic approaches are primarily aimed at rendering secondary and tertiary neurons of the retina light-sensitive in order to replace degenerate or dysfunctional photoreceptors. Such approaches are attractive because they provide a "causative gene-independent" strategy, which may prove suitable for a variety of patients with IRD. We discuss theoretical and practical considerations in the selection of optogenetic molecules, vectors, surgical approaches and review previous trials of optogenetics for vision restoration. Optogenetic approaches to vision restoration have yielded promising results in pre-clinical trials and a phase I/II clinical trial is currently underway (ClinicalTrials.gov NCT02556736). Despite the significant inroads made in recent years, the ideal optogenetic molecule, vector and surgical approach have yet to be established.

Keywords: Gene therapy; Inherited retinal disease; Optogenetics; Retinitis pigmentosa; Vision restoration.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Eye Diseases, Hereditary / therapy
Genetic Therapy*
Genetic Vectors
Humans
Retinitis Pigmentosa / therapy*
Vision Disorders / rehabilitation*

Associated data

ClinicalTrials.gov/NCT02556736