| Autor: |
Stephen C. Kolwicz, Jr., PhD, John K. Hall, PhD, Farid Moussavi-Harami, MD, Xiolan Chen, PhD, Stephen D. Hauschka, PhD, Jeffrey S. Chamberlain, PhD, Michael Regnier, PhD, Guy L. Odom, PhD |
| Jazyk: |
angličtina |
| Rok vydání: |
2019 |
| Předmět: |
|
| Zdroj: |
JACC: Basic to Translational Science, Vol 4, Iss 7, Pp 778-791 (2019) |
| Druh dokumentu: |
article |
| ISSN: |
2452-302X |
| DOI: |
10.1016/j.jacbts.2019.06.006 |
| Popis: |
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. Key Words: cardiomyopathy, diastolic dysfunction, dystrophin, ribonucleotide reductase, recombinant adeno-associated virus vectors |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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