Evaluation of MYBPC3 trans-splicing and gene replacement as therapeutic options in human iPSC-derived cardiomyocytes

Evaluation of MYBPC3 trans-splicing and gene replacement as therapeutic options in human iPSC-derived cardiomyocytes

Gene therapy is a promising option for severe forms of genetic diseases. We previously provided evidence for the feasibility of trans-splicing, exon skipping, and gene replacement in a mouse model of hypertrophic cardiomyopathy (HCM) carrying a mutation in MYBPC3, encoding cardiac myosin-binding pro...

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Bibliografiska uppgifter
Huvudupphovsmän:	Prondzynski, M, Krämer, E, Laufer, S, Shibamiya, A, Pless, O, Flenner, F, Müller, O, Münch, J, Redwood, C, Hansen, A, Patten, M, Eschenhagen, T, Mearini, G, Carrier, L
Materialtyp:	Journal article
Språk:	English
Publicerad:	Elsevier 2017

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