Proof of concept for AAV2/5-mediated gene therapy in iPSC-derived retinal pigment epithelium of a choroideremia patient.

Cereso N, Pequignot MO, Robert L, Becker F, De Luca V, Nabholz N, Rigau V, De Vos J, Hamel CP, Kalatzis V
Mol Ther Methods Clin Dev. 2014 Apr 2;1:14011. doi: 10.1038/mtm.2014.11. eCollection 2014. PubMed Article

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