Restoration of visual function by transplantation of optogenetically engineered photoreceptors

Marcela Garita-Hernandez, Maruša Lampič, Antoine Chaffiol, Laure Guibbal, Fiona Routet, Tiago Santos-Ferreira, Sylvia Gasparini, Oliver Borsch, Giuliana Gagliardi, Sacha Reichman, Serge Picaud, José-Alain Sahel, Olivier Goureau, Marius Ader, Deniz Dalkara () and Jens Duebel ()
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Marcela Garita-Hernandez: Sorbonne Université, Institut de la Vision, INSERM, CNRS
Maruša Lampič: Sorbonne Université, Institut de la Vision, INSERM, CNRS
Antoine Chaffiol: Sorbonne Université, Institut de la Vision, INSERM, CNRS
Laure Guibbal: Sorbonne Université, Institut de la Vision, INSERM, CNRS
Fiona Routet: Sorbonne Université, Institut de la Vision, INSERM, CNRS
Tiago Santos-Ferreira: CRTD/Center for Regenerative Therapies Dresden, CMCB, TU Dresden
Sylvia Gasparini: CRTD/Center for Regenerative Therapies Dresden, CMCB, TU Dresden
Oliver Borsch: CRTD/Center for Regenerative Therapies Dresden, CMCB, TU Dresden
Giuliana Gagliardi: Sorbonne Université, Institut de la Vision, INSERM, CNRS
Sacha Reichman: Sorbonne Université, Institut de la Vision, INSERM, CNRS
Serge Picaud: Sorbonne Université, Institut de la Vision, INSERM, CNRS
José-Alain Sahel: Sorbonne Université, Institut de la Vision, INSERM, CNRS
Olivier Goureau: Sorbonne Université, Institut de la Vision, INSERM, CNRS
Marius Ader: CRTD/Center for Regenerative Therapies Dresden, CMCB, TU Dresden
Deniz Dalkara: Sorbonne Université, Institut de la Vision, INSERM, CNRS
Jens Duebel: Sorbonne Université, Institut de la Vision, INSERM, CNRS

Nature Communications, 2019, vol. 10, issue 1, 1-13

Abstract: Abstract A major challenge in the treatment of retinal degenerative diseases, with the transplantation of replacement photoreceptors, is the difficulty in inducing the grafted cells to grow and maintain light sensitive outer segments in the host retina, which depends on proper interaction with the underlying retinal pigment epithelium (RPE). Here, for an RPE-independent treatment approach, we introduce a hyperpolarizing microbial opsin into photoreceptor precursors from newborn mice, and transplant them into blind mice lacking the photoreceptor layer. These optogenetically-transformed photoreceptors are light responsive and their transplantation leads to the recovery of visual function, as shown by ganglion cell recordings and behavioral tests. Subsequently, we generate cone photoreceptors from human induced pluripotent stem cells, expressing the chloride pump Jaws. After transplantation into blind mice, we observe light-driven responses at the photoreceptor and ganglion cell levels. These results demonstrate that structural and functional retinal repair is possible by combining stem cell therapy and optogenetics.

Date: 2019
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DOI: 10.1038/s41467-019-12330-2

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