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Let there be light: gene and cell therapy for blindness.

Abstract : Retinal degenerative diseases are a leading cause of irreversible blindness. Retinal cell death is the main cause of vision loss in genetic disorders such as retinitis pigmentosa, Stargardt disease and Leber congenital amaurosis, as well as in complex age-related diseases such as age-related macular degeneration (AMD). For these blinding conditions, gene and cell therapy approaches offer therapeutic intervention at various disease stages. The present review outlines recent advances in therapies for retinal degenerative disease, focusing on the progress and challenges in the development and clinical translation of gene and cell therapies. A significant body of preclinical evidence and initial clinical results pave the way for further development of these cutting edge treatments for patients with retinal degenerative disorders.
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Submitted on : Tuesday, January 19, 2016 - 11:20:55 AM
Last modification on : Sunday, June 26, 2022 - 9:51:07 AM
Long-term archiving on: : Friday, November 11, 2016 - 11:25:14 AM


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  • HAL Id : inserm-01258538, version 1
  • PUBMED : 26751519


Deniz Dalkara, Olivier Goureau, Katia Marazova, José-Alain Sahel. Let there be light: gene and cell therapy for blindness.. Human Gene Therapy, Mary Ann Liebert, 2016, 27 (2), pp.134-47. ⟨inserm-01258538⟩



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