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review · Human Gene Therapy · 2016

Let There Be Light: Gene and Cell Therapy for Blindness

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

Cited by 123Abstract and details only

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.

Cite this paper

APA 7

Dalkara, D., Goureau, O., Marazova, K., & Sahel, J. (2016). Let There Be Light: Gene and Cell Therapy for Blindness. Human Gene Therapy, 27(2), 134–147. https://doi.org/10.1089/hum.2015.147

MLA 9

Dalkara, Deniz, et al. “Let There Be Light: Gene and Cell Therapy for Blindness.” Human Gene Therapy, vol. 27, no. 2, 2016, pp. 134–47, https://doi.org/10.1089/hum.2015.147.

Chicago

Dalkara, Deniz, Olivier Goureau, Katia Marazova, and José‐Alain Sahel. 2016. “Let There Be Light: Gene and Cell Therapy for Blindness.” Human Gene Therapy 27 (2): 134–47. https://doi.org/10.1089/hum.2015.147.

Harvard

Dalkara, D. et al. (2016) ‘Let There Be Light: Gene and Cell Therapy for Blindness’, Human Gene Therapy, 27(2), pp. 134–147. Available at: https://doi.org/10.1089/hum.2015.147.

BibTeX
@article{dalkara2016there,
  title = {{Let There Be Light: Gene and Cell Therapy for Blindness}},
  author = {Dalkara, Deniz and Goureau, Olivier and Marazova, Katia and Sahel, José‐Alain},
  journal = {Human Gene Therapy},
  volume = {27},
  number = {2},
  pages = {134--147},
  year = {2016},
  doi = {10.1089/hum.2015.147}
}

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