Article
Multimodal assessment of choroideremia patients defines pre-treatment characteristics.
Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie - 1 Dec 2015
Seitz Immanuel P, Zhour Ahmad, Kohl Susanne, Llavona Pablo, Peter Tobias, Wilhelm Barbara, Zrenner Eberhart, Ueffing Marius, Bartz-Schmidt Karl Ulrich, Fischer M Dominik
Abstract excerpt
PURPOSE: Choroideremia (CHM) is a X-chromosomal disorder leading to blindness by progressive degeneration of choroid, retinal pigment epithelium (RPE), and retinal neurons. A current clinical gene therapy trial (NCT01461213) showed promising safety and efficacy data in a carefully selected patient population. The present study was performed to shed light on pre-treatment characteristics of a larger cohort of CHM...
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