Title : Dual DNA Transfection Using 1,6-Hexanedithiol-Conjugated Maleimide-Functionalized PU-PEI600 For Gene Correction in a Patient iPSC-Derived Fabry Cardiomyopathy Model.

Pub. Date : 2021

PMID : 34422789






1 Functional Relationships(s)
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1 This FC in vitro model recapitulated several clinical FC features, including cardiomyocyte hypertrophy and lysosomal globotriaosylceramide (Gb3) deposition. globotriaosylceramide alpha 1,4-galactosyltransferase (P blood group) Homo sapiens