Document Detail


Potential of gene therapy as a treatment for heart failure.
MedLine Citation:
PMID:  23281410     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
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.
Authors:
Roger J Hajjar
Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural     Date:  2013-01-02
Journal Detail:
Title:  The Journal of clinical investigation     Volume:  123     ISSN:  1558-8238     ISO Abbreviation:  J. Clin. Invest.     Publication Date:  2013 Jan 
Date Detail:
Created Date:  2013-01-02     Completed Date:  2013-03-11     Revised Date:  2014-01-09    
Medline Journal Info:
Nlm Unique ID:  7802877     Medline TA:  J Clin Invest     Country:  United States    
Other Details:
Languages:  eng     Pagination:  53-61     Citation Subset:  AIM; IM    
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MeSH Terms
Descriptor/Qualifier:
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*
Grant Support
ID/Acronym/Agency:
HHSN26820100045C//PHS HHS; HL088434/HL/NHLBI NIH HHS; P20HL100396/HL/NHLBI NIH HHS; P50HL 112324/HL/NHLBI NIH HHS; R01 HL093183/HL/NHLBI NIH HHS
Chemical
Reg. No./Substance:
0/Muscle Proteins; EC 3.6.3.8/Sarcoplasmic Reticulum Calcium-Transporting ATPases
Comments/Corrections

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