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Let There Be Light: Gene and Cell Therapy for Blindness.

作者信息

Dalkara Deniz, Goureau Olivier, Marazova Katia, Sahel José-Alain

机构信息

1 Sorbonne Universités, UPMC Université Paris 06, INSERM, CNRS, Institut de la Vision , France .

2 Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts , DHU Sight Restore, INSERM-DHOS CIC 1423, France .

出版信息

Hum Gene Ther. 2016 Feb;27(2):134-47. doi: 10.1089/hum.2015.147.


DOI:10.1089/hum.2015.147
PMID:26751519
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4779297/
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. For these blinding conditions, gene and cell therapy approaches offer therapeutic intervention at various disease stages. The present review outlines 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.

摘要

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[6]
Stargardt macular dystrophy and therapeutic approaches.

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[7]
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[8]
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[9]
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[10]
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本文引用的文献

[1]
Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs.

Gene Ther. 2016-4

[2]
Gene Therapy for Leber Hereditary Optic Neuropathy: Initial Results.

Ophthalmology. 2016-3

[3]
Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease.

Hum Mol Genet. 2015-12-1

[4]
Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness.

Hum Mol Genet. 2015-11-1

[5]
Halting progressive neurodegeneration in advanced retinitis pigmentosa.

J Clin Invest. 2015-9

[6]
iPS Cells for Modelling and Treatment of Retinal Diseases.

J Clin Med. 2014-12-19

[7]
Restoration of Vision with Ectopic Expression of Human Rod Opsin.

Curr Biol. 2015-8-17

[8]
Plasticity of the human visual system after retinal gene therapy in patients with Leber's congenital amaurosis.

Sci Transl Med. 2015-7-15

[9]
Stem cells in age-related macular degeneration and Stargardt's macular dystrophy - Authors' reply.

Lancet. 2015-7-4

[10]
Long-Term Results from an Epiretinal Prosthesis to Restore Sight to the Blind.

Ophthalmology. 2015-8

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