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Potential of gene therapy as a treatment for heart failure.

Abstract
Advances in understanding the molecular basis of myocardial dysfunction, together with the evolution of increasingly efficient gene transfer technology, make gene-based therapy a promising treatment option for heart conditions. Cardiovascular gene therapy has benefitted from recent advancements in vector technology, design, and delivery modalities. There is a critical need to explore new therapeutic approaches in heart failure, and gene therapy has emerged as a viable alternative. Advances in understanding of the molecular basis of myocardial dysfunction, together with the development of increasingly efficient gene transfer technology, has placed heart failure within reach of gene-based therapy. The recent successful and safe completion of a phase 2 trial targeting the cardiac sarcoplasmic/endoplasmic reticulum Ca2+ ATPase pump (SERCA2a) has the potential to open a new era for gene therapy for heart failure.
AuthorsRoger J Hajjar
JournalThe Journal of clinical investigation (J Clin Invest) Vol. 123 Issue 1 Pg. 53-61 (Jan 2013) ISSN: 1558-8238 [Electronic] United States
PMID23281410 (Publication Type: Journal Article, Research Support, N.I.H., Extramural)
Chemical References
  • Muscle Proteins
  • Sarcoplasmic Reticulum Calcium-Transporting ATPases
Topics
  • Gene Transfer Techniques
  • Genetic Therapy (methods)
  • Heart Failure (genetics, metabolism, pathology, therapy)
  • Muscle Proteins (biosynthesis, genetics)
  • Myocardium (metabolism, pathology)
  • Sarcoplasmic Reticulum Calcium-Transporting ATPases (biosynthesis, genetics)

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