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Optogenetic therapy for retinitis pigmentosa

Gene Therapy volume 19, pages 169–175 (2012)Cite this article

Abstract

Retinitis pigmentosa (RP) refers to a diverse group of progressive, hereditary diseases of the retina that lead to incurable blindness and affect two million people worldwide. Artificial photoreceptors constructed by gene delivery of light-activated channels or pumps (‘optogenetic tools’) to surviving cell types in the remaining retinal circuit has been shown to restore photosensitivity in animal models of RP at the level of the retina and cortex as well as behaviorally. The translational potential of this optogenetic approach has been evaluated using in vitro studies involving post-mortem human retinas. Here, we review recent developments in this expanding field and discuss the potential and limitations of optogenetic engineering for the treatment of RP.

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Authors and Affiliations

  1. Neural Circuit Laboratories, Friedrich Miescher Institute for Biomedical Research, Basel, Switzerland

    V Busskamp & B Roska

  2. NCCR Frontiers in Genetics Program, University of Geneva, Geneva, Switzerland,

    V Busskamp

  3. Inserm, UMR_S968, Institut de la Vision, Paris, France

    S Picaud & J A Sahel

  4. UPMC Univ Paris 06, UMR_S968, Institut de la Vision, Paris, France

    S Picaud & J A Sahel

  5. CNRS, UMR_7210, Paris, France

    S Picaud & J A Sahel

  6. Fondation Ophtalmologique Adolphe de Rothschild, Paris, France

    S Picaud & J A Sahel

  7. Centre d’Investigation Clinique 503, Inserm-Centre Hospitalier National d’Ophtalmologie des, Quinze-Vingts, Paris, France

    J A Sahel

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  1. V Busskamp
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  2. S Picaud
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  3. J A Sahel
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  4. B Roska
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Correspondence to B Roska.

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Busskamp, V., Picaud, S., Sahel, J. et al. Optogenetic therapy for retinitis pigmentosa. Gene Ther 19, 169–175 (2012). https://doi.org/10.1038/gt.2011.155

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