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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Vydáno v:Mol Ther Nucleic Acids
Hlavní autoři: Prondzynski, Maksymilian, Krämer, Elisabeth, Laufer, Sandra D., Shibamiya, Aya, Pless, Ole, Flenner, Frederik, Müller, Oliver J., Münch, Julia, Redwood, Charles, Hansen, Arne, Patten, Monica, Eschenhagen, Thomas, Mearini, Giulia, Carrier, Lucie
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2017
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5458066/
https://ncbi.nlm.nih.gov/pubmed/28624223
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.05.008
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