Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice.

Gedicke-Hornung, Christina

Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice. [electronic resource] - EMBO molecular medicine Jul 2013 - 1128-45 p. digital

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

1757-4684

10.1002/emmm.201202168 doi


Adenoviridae--genetics
Alternative Splicing
Animals
Cardiomyopathy, Hypertrophic--genetics
Carrier Proteins--genetics
Exons
Gene Knock-In Techniques
Genetic Therapy
HEK293 Cells
Heart--physiopathology
Humans
Hypertrophy, Left Ventricular--prevention & control
Mice
Mutation
Myocardium--metabolism
Oligoribonucleotides, Antisense--administration & dosage
Protein Isoforms--genetics
RNA, Messenger--genetics
RNA, Small Nuclear--administration & dosage
Transduction, Genetic