File(s) not publicly available

Gene therapy and the adeno-associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: trials, future directions and safety considerations

journal contribution
posted on 07.08.2019, 00:00 by Charmaine Ramlogan-SteelCharmaine Ramlogan-Steel, A Murali, S Andrzejewski, B Dhungel, Jason SteelJason Steel, CJ Layton
Voretigene neparvovec‐rzyl was recently approved for the treatment of Leber Congenital Amaurosis, and the use of gene therapy for eye disease is attracting even greater interest. The eye has immune privileged status, is easily accessible, requires a reduced dosage of therapy due to its size, and is highly compartmentalized, significantly reducing systemic spread. Adeno‐associated virus (AAV), with its low pathogenicity, prolonged expression profile and ability to transduce multiple cell types, has become the leading gene therapy vector. Target diseases have moved beyond currently untreatable inherited dystrophies to common, partially treatable acquired conditions such as exudative AMD and glaucoma, but use of the technology in these conditions imposes added obligations for caution in vector design. This review discusses the current status of AAV gene therapy trials in genetic and acquired ocular diseases, and explores new scientific developments which could help ensure effective and safe use of the therapy in the future.

History

Volume

47

Issue

4

Start Page

521

End Page

536

Number of Pages

16

eISSN

1442-9071

ISSN

1442-6404

Publisher

Wiley-Blackwell, Australia

Peer Reviewed

Yes

Open Access

No

Acceptance Date

15/10/2018

External Author Affiliations

Translational Research Institute, Woolloongabba, QLD, Australia; University of Queensland

Era Eligible

Yes

Journal

Clinical and Experimental Ophthalmology