Evaluation of MYBPC3 trans-Splicing and Gene Replacement as Therapeutic Options in Human iPSC-Derive...
Evaluation of MYBPC3 trans-Splicing and Gene Replacement as Therapeutic Options in Human iPSC-Derived Cardiomyocytes
About this item
Full title
Author / Creator
Publisher
United States: Elsevier Inc
Journal title
Language
English
Formats
Publication information
Publisher
United States: Elsevier Inc
Subjects
More information
Scope and Contents
Contents
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 protein C (cMyBP-C). Here we used human induced pluri...
Alternative Titles
Full title
Evaluation of MYBPC3 trans-Splicing and Gene Replacement as Therapeutic Options in Human iPSC-Derived Cardiomyocytes
Authors, Artists and Contributors
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_285f249a121446408d9780a45f0af2f9
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_285f249a121446408d9780a45f0af2f9
Other Identifiers
ISSN
2162-2531
E-ISSN
2162-2531
DOI
10.1016/j.omtn.2017.05.008
