Allele-Specific Silencing of Mutant Myh6 Transcripts in Mice Suppresses Hypertrophic Cardiomyopathy
Why the study?
Does an RNA interference (RNAi) cassette delivered by an adeno-associated virus vector prevent hypertrophy and myocardial fibrosis in MHC(403/+) mice?
Population
MHC(403/+) mice expressing the HCM-causing cardiac MHC gene (Myh6) R403Q mutation
Design
Preclinical
Follow-up
at least 6 months
Authors
Discussion
Loading...
Member takes
Implication
Hypothesis-generating for AAV-RNAi in genetic HCM; requires validation before human translation.
Structured PICO
Does an RNA interference (RNAi) cassette delivered by an adeno-associated virus vector prevent hypertrophy and myocardial fibrosis in MHC(403/+) mice?
P
Population
MHC(403/+) mice expressing the HCM-causing cardiac MHC gene (Myh6) R403Q mutation
I
Intervention
RNA interference (RNAi) cassette delivered by an adeno-associated virus vector
O
Outcome
Development of hypertrophy and myocardial fibrosissurrogate
Allele-specific silencing of the mutant Myh6 transcript via AAV-delivered RNAi prevents the development of hypertrophic cardiomyopathy phenotypes in a mouse model.
Cite This Study
Jiang et al. (2013) studied this question.