Gene therapy rescues cardiac dysfunction in Duchenne muscular dystrophy mice by elevating cardiomyocyte deoxy-adenosine triphosphate
SC Kolwicz, JK Hall, F Moussavi-Harami… - JACC: Basic to …, 2019 - jacc.org
JACC: Basic to Translational Science, 2019•jacc.org
Mutations in the gene encoding for dystrophin leads to structural and functional deterioration
of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy
(DMD) patients. Administration of recombinant adeno-associated viral vectors delivering
microdystrophin or ribonucleotide reductase (RNR), under muscle-specific regulatory
control, rescues both baseline and high workload-challenged hearts in an aged, DMD
mouse model. However, only RNR treatments improved both systolic and diastolic function …
of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy
(DMD) patients. Administration of recombinant adeno-associated viral vectors delivering
microdystrophin or ribonucleotide reductase (RNR), under muscle-specific regulatory
control, rescues both baseline and high workload-challenged hearts in an aged, DMD
mouse model. However, only RNR treatments improved both systolic and diastolic function …
Summary
Mutations in the gene encoding for dystrophin leads to structural and functional deterioration of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy (DMD) patients. Administration of recombinant adeno-associated viral vectors delivering microdystrophin or ribonucleotide reductase (RNR), under muscle-specific regulatory control, rescues both baseline and high workload-challenged hearts in an aged, DMD mouse model. However, only RNR treatments improved both systolic and diastolic function under those conditions. Cardiac-specific recombinant adeno-associated viral treatment of RNR holds therapeutic promise for improvement of cardiomyopathy in DMD patients.
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