Why the study?
Does transgenic over-expression of cardiac α-actin improve survival in mouse models of severe dominant ACTA1 disease?
Population
Mouse models of severe dominant ACTA1 disease (ACTA1(D286G). Acta1(+/-) mice and Acta1(H40Y) mice)
Comparison
Transgenic over-expression of cardiac α-actin in… vs Mice without the cardiac α-actin transgene
Design
Preclinical
Follow-up
Up to 5 months of age
Key result
Transgenic over-expression of cardiac α-actin reduced lethality before 30 days of age from ~59% to ~12% in the ACTA1(D286G) mouse model of dominant ACTA1 disease.
Authors
Loading...
May support α-actin augmentation in ACTA1 models; leaves open human translation and broader applicability.
Does transgenic over-expression of cardiac α-actin improve survival in mouse models of severe dominant ACTA1 disease?
Cardiac α-actin over-expression reduces lethality in some, but not all, mouse models of dominant ACTA1 disease, suggesting potential therapeutic value.
Absolute Event Rate: 12% vs 59%
Ravenscroft et al. (2013) studied Dominant ACTA1 disease. Transgenic over-expression of cardiac α-actin vs. Control mice without the transgene was evaluated on Lethality before 30 days of age (in ACTA1(D286G) model). Transgenic over-expression of cardiac α-actin reduced lethality before 30 days of age from ~59% to ~12% in the ACTA1(D286G) mouse model of dominant ACTA1 disease.