Sustaining cardiac claudin-5 levels prevents functional hallmarks of cardiomyopathy in a muscular dystrophy mouse model

Mol Ther. 2012 Jul;20(7):1378-83. doi: 10.1038/mt.2012.81. Epub 2012 May 1.

Abstract

Identification of new molecular targets in heart failure could ultimately have a substantial positive impact on both the health and financial aspects of treating the large heart failure population. We originally identified reduced levels of the cell junction protein claudin-5 specifically in heart in the dystrophin/utrophin-deficient (Dmd(mdx);Utrn(-/-)) mouse model of muscular dystrophy and cardiomyopathy, which demonstrates physiological hallmarks of heart failure. We then showed that at least 60% of cardiac explant samples from patients with heart failure resulting from diverse etiologies also have reduced claudin-5 levels. These claudin-5 reductions were independent of changes in other cell junction proteins previously linked to heart failure. The goal of this study was to determine whether sustaining claudin-5 levels is sufficient to prevent the onset of histological and functional indicators of heart failure. Here, we show the proof-of-concept rescue experiment in the Dmd(mdx);Utrn(-/-) model, in which claudin-5 reductions were originally identified. Expression of claudin-5 4 weeks after a single administration of recombinant adeno-associated virus (rAAV) containing a claudin-5 expression cassette prevented the onset of physiological hallmarks of cardiomyopathy and improved histological signs of cardiac damage. This experiment demonstrates that claudin-5 may represent a novel treatment target for prevention of heart failure.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cardiomyopathies / prevention & control*
  • Claudin-5 / genetics*
  • Claudin-5 / metabolism*
  • Dependovirus / genetics
  • Disease Models, Animal
  • Dystrophin / genetics
  • Gene Transfer Techniques
  • Heart Failure / genetics
  • Heart Failure / prevention & control*
  • Mice
  • Mice, Inbred C57BL
  • Mice, Transgenic
  • Muscular Dystrophy, Animal / genetics
  • Muscular Dystrophy, Animal / metabolism*
  • Myocardium / metabolism*
  • Utrophin / deficiency
  • Utrophin / genetics
  • Utrophin / metabolism

Substances

  • Claudin-5
  • Cldn5 protein, mouse
  • Dystrophin
  • Utrn protein, mouse
  • Utrophin
  • apo-dystrophin 1