posted on 2023-05-22, 23:54authored bySimunovic, MP, Shen, W, John LinJohn Lin, Protti, DA, Lisowski, L, Gillies, MC
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.
Funding
National Health & Medical Research Council
History
Publication title
Experimental Eye Research
Volume
178
Pagination
15-26
ISSN
0014-4835
Department/School
Tasmanian School of Medicine
Publisher
Academic Press Ltd Elsevier Science Ltd
Place of publication
24-28 Oval Rd, London, England, Nw1 7Dx
Repository Status
Restricted
Socio-economic Objectives
Clinical health not elsewhere classified; Other health not elsewhere classified; Expanding knowledge in the biological sciences