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Pharmaceutics
Volume 12
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10.3390/pharmaceutics12090836
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Article

Transferrin Non-Viral Gene Therapy for Treatment of Retinal Degeneration

by
Karine Bigot
1,
Pauline Gondouin
1,
Romain Bénard
1,
Pierrick Montagne
1,
Jenny Youale
1,2,
Marie Piazza
1,
Emilie Picard
2,
Thierry Bordet
1,* and
Francine Behar-Cohen
2,3,*
1
Eyevensys, Biopark, 11 rue Watt, 75013 Paris, France
2
Centre de Recherche des Cordeliers, INSERM, Sorbonne Université, USPC, Université Paris Descartes, Team 17, 75006 Paris, France
3
Ophtalmopole, Cochin Hospital, AP-HP, Assistance Publique Hôpitaux de Paris, 24 rue du Faubourg Saint-Jacques, 75014 Paris, France
*
Authors to whom correspondence should be addressed.
Pharmaceutics 2020, 12(9), 836; https://doi.org/10.3390/pharmaceutics12090836
Submission received: 7 August 2020 / Revised: 26 August 2020 / Accepted: 28 August 2020 / Published: 1 September 2020
(This article belongs to the Special Issue Ophthalmic Drug Delivery)
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Abstract

Dysregulation of iron metabolism is observed in animal models of retinitis pigmentosa (RP) and in patients with age-related macular degeneration (AMD), possibly contributing to oxidative damage of the retina. Transferrin (TF), an endogenous iron chelator, was proposed as a therapeutic candidate. Here, the efficacy of TF non-viral gene therapy based on the electrotransfection of pEYS611, a plasmid encoding human TF, into the ciliary muscle was evaluated in several rat models of retinal degeneration. pEYS611 administration allowed for the sustained intraocular production of TF for at least 3 and 6 months in rats and rabbits, respectively. In the photo-oxidative damage model, pEYS611 protected both retinal structure and function more efficiently than carnosic acid, a natural antioxidant, reduced microglial infiltration in the outer retina and preserved the integrity of the outer retinal barrier. pEYS611 also protected photoreceptors from N-methyl-N-nitrosourea-induced apoptosis. Finally, pEYS611 delayed structural and functional degeneration in the RCS rat model of RP while malondialdehyde (MDA) ocular content, a biomarker of oxidative stress, was decreased. The neuroprotective benefits of TF non-viral gene delivery in retinal degenerative disease models further validates iron overload as a therapeutic target and supports the continued development of pEY611 for treatment of RP and dry AMD.
Keywords: iron; retinal degeneration; age-related macular degeneration; retinitis pigmentosa; transferrin; gene therapy; plasmid electrotransfection iron; retinal degeneration; age-related macular degeneration; retinitis pigmentosa; transferrin; gene therapy; plasmid electrotransfection

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MDPI and ACS Style

Bigot, K.; Gondouin, P.; Bénard, R.; Montagne, P.; Youale, J.; Piazza, M.; Picard, E.; Bordet, T.; Behar-Cohen, F. Transferrin Non-Viral Gene Therapy for Treatment of Retinal Degeneration. Pharmaceutics 2020, 12, 836. https://doi.org/10.3390/pharmaceutics12090836

AMA Style

Bigot K, Gondouin P, Bénard R, Montagne P, Youale J, Piazza M, Picard E, Bordet T, Behar-Cohen F. Transferrin Non-Viral Gene Therapy for Treatment of Retinal Degeneration. Pharmaceutics. 2020; 12(9):836. https://doi.org/10.3390/pharmaceutics12090836

Chicago/Turabian Style

Bigot, Karine, Pauline Gondouin, Romain Bénard, Pierrick Montagne, Jenny Youale, Marie Piazza, Emilie Picard, Thierry Bordet, and Francine Behar-Cohen. 2020. "Transferrin Non-Viral Gene Therapy for Treatment of Retinal Degeneration" Pharmaceutics 12, no. 9: 836. https://doi.org/10.3390/pharmaceutics12090836

APA Style

Bigot, K., Gondouin, P., Bénard, R., Montagne, P., Youale, J., Piazza, M., Picard, E., Bordet, T., & Behar-Cohen, F. (2020). Transferrin Non-Viral Gene Therapy for Treatment of Retinal Degeneration. Pharmaceutics, 12(9), 836. https://doi.org/10.3390/pharmaceutics12090836

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