Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives

Thierry Bordet (1) , Francine Behar-Cohen (2, 3, 4, 5)

1 Eyevensys S.A.S. [Paris, France]
2 CRC (UMR_S_1138 / U1138) - Centre de Recherche des Cordeliers
3 USPC - Université Sorbonne Paris Cité
4 Hôpital Cochin [AP-HP]
5 UPMC - Université Pierre et Marie Curie - Paris 6

Ocular gene therapy has entered into clinical practice. Although viral vectors are currently the best option to replace and/or correct genes, the optimal method to deliver these treatments to the retinal pigment epithelial (RPE) cells and/or photoreceptor cells remains to be improved to increase transduction efficacy and reduce iatrogenic risks. Beyond viral-mediated gene replacement therapies, nonviral 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 can address more common nongenetic retinal diseases, such as age-related macular degeneration (AMD). Here, we review current gene therapy strategies for ocular diseases, with a focus on clinical stage products.

Domaines: Organes des sens

Thierry Bordet, Francine Behar-Cohen. Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives. Drug Discovery Today Biosilico, 2019, 24 (8), pp.1685-1693. ⟨10.1016/j.drudis.2019.05.038⟩. ⟨hal-02303632⟩