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Q36433790-44E78CE8-88AF-4405-BF0F-7E7F1F8A995A
Q36433790-44E78CE8-88AF-4405-BF0F-7E7F1F8A995A
BestRank
Statement
http://www.wikidata.org/entity/statement/Q36433790-44E78CE8-88AF-4405-BF0F-7E7F1F8A995A
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.
P2860
Q36433790-44E78CE8-88AF-4405-BF0F-7E7F1F8A995A
BestRank
Statement
http://www.wikidata.org/entity/statement/Q36433790-44E78CE8-88AF-4405-BF0F-7E7F1F8A995A
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wasDerivedFrom
0a07edfef875aa2724d7772d766d6b5af023f6a4
P2860
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial