Ocular gene therapy for choroideremia: clinical trials and future perspectives
Introduction: Gene therapy offers the potential for targeted replacement of single gene defects in inherited retinal degenerations. Areas covered: Choroideremia is an X-linked blinding retinal disease resulting from deficiency of the CHM gene product, REP1. The disease represents an ideal target for retinal gene therapy, as it is readily diagnosed in the clinic, relatively homogenous in phenotype and slow progressing, thereby providing a wide therapeutic win... Expand abstract
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- Xue and MacLaren
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This article was originally published with errors. This version has been amended. Please see Erratum (http://dx.doi.org/10.1080/17469899.2018.1484136)
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