| Literature DB >> 31998848 |
Stephen C Kolwicz1, John K Hall2, Farid Moussavi-Harami3, Xiolan Chen4,5, Stephen D Hauschka4,5, Jeffrey S Chamberlain2,4,5, Michael Regnier5,6,7, Guy L Odom2,5,7.
Abstract
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.Entities:
Keywords: CK8, miniaturized murine creatine kinase regulatory cassette; CMV, cytomegalovirus; DMD, Duchenne muscular dystrophy; RNR, ribonucleotide reductase; cTnT, cardiac troponin T; cardiomyopathy; dADP, deoxy-adenosine diphosphate; dATP, deoxy-adenosine triphosphate; diastolic dysfunction; dystrophin; mdx, mouse muscular dystrophy model; rAAV, recombinant adeno-associated viral vector; recombinant adeno-associated virus vectors; ribonucleotide reductase; μDys, microdystrophin
Year: 2019 PMID： 31998848 PMCID： PMC6978556 DOI： 10.1016/j.jacbts.2019.06.006
Source DB: PubMed Journal: JACC Basic Transl Sci ISSN： 2452-302X