PKC inhibition ameliorates the cardiac phenotype in a mouse model of myotonic dystrophy type 1
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Why the study?
Does PKC inhibition improve survival and ameliorate the cardiac phenotype in a mouse model of myotonic dystrophy type 1?
Population
Tamoxifen-inducible heart-specific myotonic dystrophy type 1 mouse model.
Comparison
PKC inhibitor 6 mg/kg/day administered… vs Mock control receiving daily saline injections.
Design
Preclinical
Follow-up
Up to 43 days
Authors
Discussion
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Implication
PKC inhibition may improve survival in DM1 mouse models; leaves open human translation pending clinical trials.
Structured PICO
Does PKC inhibition improve survival and ameliorate the cardiac phenotype in a mouse model of myotonic dystrophy type 1?
P
Population
Tamoxifen-inducible heart-specific myotonic dystrophy type 1 (DM1) mouse model (EpA960/MCM bitransgenic mice expressing expanded CUG RNA).
I
Intervention
PKC inhibitor (Ro-31-8220 or Ro-32-0432) 6 mg/kg/day administered intraperitoneally for up to 24-43 days.
C
Comparator
Mock control receiving daily saline injections.
O
Outcome
Mortality within 3 weeks of RNA induction.hard clinical
Pharmacological blockade of PKC activity mitigates the cardiac phenotype and significantly improves survival in a mouse model of myotonic dystrophy type 1.
Cite This Study
Wang et al. (2009) studied this question.
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