Key result
Systemic injection of AAV9-C0C2 significantly improved ejection fraction to 52.24% compared with 40.07% in control mice and reduced histopathologic signs of cardiomyopathy.
Why the study?
It was unknown whether cMyBPC N-terminal domains can regulate in vivo myocardial function and recapitulate full-length cMyBPC in rescuing cardiac function in HCM.
Does AAV9 gene transfer of cMyBPC N-terminal domains (C0C2) improve cardiac function and reduce cardiomyopathy in a cMyBPC-deficient mouse model of HCM?
Population
cMyBPC-null mouse model of HCM treated at neonatal day 1
Comparison
AAV9-C0C2 vs AAV9-FL gene replacement vs control cMyBPC-/-
Authors
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Should not change clinical practice; hypothesis-generating for AAV9 gene therapy in cMyBPC-deficient HCM.
Does AAV9 gene transfer of cMyBPC N-terminal domains (C0C2) improve cardiac function and reduce cardiomyopathy in a cMyBPC-deficient mouse model of HCM?
Absolute Event Rate: 52.24% vs 40.07%
p-value: p=<0.05
Gene transfer of the N-terminal domains of cMyBPC (C0C2) is sufficient to rescue biomechanical defects and ameliorate the hypertrophic cardiomyopathy phenotype in a cMyBPC-deficient mouse model.
Li et al. (2020) studied Hypertrophic cardiomyopathy (cMyBPC deficiency). AAV9-C0C2 gene transfer vs. AAV9-GFP (vehicle control) was evaluated on Ejection fraction at 6 weeks (p=<0.05). Systemic injection of AAV9-C0C2 significantly improved ejection fraction to 52.24% compared with 40.07% in control mice and reduced histopathologic signs of cardiomyopathy.
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