Using patient-specific induced pluripotent stem cells to interrogate the pathogenicity of a novel retinal pigment epithelium-specific 65 kDa cryptic splice site mutation and confirm eligibility for enrollment into a clinical gene augmentation trial

Title
Using patient-specific induced pluripotent stem cells to interrogate the pathogenicity of a novel retinal pigment epithelium-specific 65 kDa cryptic splice site mutation and confirm eligibility for enrollment into a clinical gene augmentation trial
Authors
Keywords
-
Journal
Translational Research
Volume 166, Issue 6, Pages 740-749.e1
Publisher
Elsevier BV
Online
2015-08-29
DOI
10.1016/j.trsl.2015.08.007

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