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.
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Authors | Roger J Hajjar |
Journal | The Journal of clinical investigation
(J Clin Invest)
Vol. 123
Issue 1
Pg. 53-61
(Jan 2013)
ISSN: 1558-8238 [Electronic] United States |
PMID | 23281410
(Publication Type: Journal Article, Research Support, N.I.H., Extramural)
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Chemical References |
- Muscle Proteins
- Sarcoplasmic Reticulum Calcium-Transporting ATPases
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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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