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Ocular gene therapies in clinical practice: Viral vectors and non‐viral alternatives
Author(s) -
BeharCohen Francine
Publication year - 2019
Publication title -
acta ophthalmologica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.534
H-Index - 87
eISSN - 1755-3768
pISSN - 1755-375X
DOI - 10.1111/j.1755-3768.2019.8233
Subject(s) - genetic enhancement , viral vector , macular degeneration , gene delivery , gene , clinical practice , retinal , disease , bioinformatics , medicine , retinal pigment epithelium , biology , computational biology , ophthalmology , pathology , genetics , family medicine , recombinant dna
Ocular gene therapy has entered into the clinical practice. While viral vectors are today the best option to replace/correct genes, the optimal method to deliver these treatments to the retinal pigment epithelial (RPE) cells and / or to the photoreceptor cells, non‐viral gene delivery approaches offer the promise of sustained fine‐tuned expression of secreted therapeutic proteins that can be adapted to the evolving stage of the disease course and that can address more common non‐genetic retinal diseases like age‐related macular degeneration (AMD). Current gene therapy strategies for ocular diseases will be reviewed here with a focus on clinical stage products. Conflict of interest : Patent, consultancy and founder of Eyevensys.

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