Journal
JOURNAL OF CLINICAL INVESTIGATION
Volume 123, Issue 1, Pages 53-61Publisher
AMER SOC CLINICAL INVESTIGATION INC
DOI: 10.1172/JCI62837
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Funding
- NIH [R01 HL093183, HL088434, P20HL100396]
- Program of Excellence in Nanotechnology [HHSN26820100045c]
- [P50HL 112324]
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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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