The effect of PTC124 on choroideremia fibroblasts and iPSC-derived RPE raises considerations for therapy-Reference-Cited by-同舟云学术

The effect of PTC124 on choroideremia fibroblasts and iPSC-derived RPE raises considerations for therapy

Published:2018-05-29 Issue:1 Volume:8 Page:
ISSN:2045-2322
Container-title:Scientific Reports
language:en
Short-container-title:Sci Rep

Author:

Torriano Simona,Erkilic Nejla,Baux David^ORCID,Cereso Nicolas,De Luca Valerie,Meunier Isabelle^ORCID,Moosajee Mariya,Roux Anne-Francoise,Hamel Christian P.,Kalatzis Vasiliki^ORCID

Publisher

Springer Science and Business Media LLC

Subject

Multidisciplinary

Link

http://www.nature.com/articles/s41598-018-26481-7.pdf

Reference53 articles.

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2. Bainbridge, J. W. et al. Effect of gene therapy on visual function in Leber’s congenital amaurosis. N. Engl. J. Med. 358, 2231–2239 (2008).

3. Maguire, A. M. et al. Safety and efficacy of gene transfer for Leber’s congenital amaurosis. N. Engl. J. Med. 358, 2240–2248 (2008).

4. MacLaren, R. E. et al. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet 383, 1129–1137, https://doi.org/10.1016/S0140-6736(13)62117-0 (2014).

5. Ghazi, N. G. et al. Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial. Hum. Genet. 135, 327–343, https://doi.org/10.1007/s00439-016-1637-y (2016).

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