State of the art and perspectives of gene therapy in heart failure. A scientific statement of the Heart Failure Association of the ESC, the ESC Council on Cardiovascular Genomics and the ESC Working Group on Myocardial & Pericardial Diseases

Tools

Tools

Tools

Refer BibTeX RefWorks HTML Citation Atom Simple Metadata ASCII Citation Dublin Core MPEG-21 DIDL EndNote Bibliography PubMed-IDs Reference Manager JSON

Preview

PDF (Original Article) - Requires a PDF viewer such as GSview, Xpdf or Adobe Acrobat Reader 1MB

Item Type:	Article
Title:	State of the art and perspectives of gene therapy in heart failure. A scientific statement of the Heart Failure Association of the ESC, the ESC Council on Cardiovascular Genomics and the ESC Working Group on Myocardial & Pericardial Diseases
Creators Name:	Van Linthout, S., Stellos, K., Giacca, M., Bertero, E., Cannata, A., Carrier, L., Garcia-Pavia, P., Ghigo, A., González, A., Haugaa, K.H., Imazio, M., Lopes, L.R., Most, P., Pollesello, P., Schunkert, H., Streckfuss-Bömeke, K., Thum, T., Tocchetti, C.G., Tschöpe, C., van der Meer, P., van Rooij, E., Metra, M., Rosano, G.M.C. and Heymans, S.
Abstract:	Gene therapy has recently become a reality in the treatment of cardiovascular diseases. Strategies to modulate gene expression using antisense oligonucleotides or small interfering RNA are proving to be safe and effective in the clinic. Adeno-associated viral vector-based gene delivery and CRISPR-Cas9-based genome editing have emerged as efficient strategies for gene delivery and repair in humans. Overall, gene therapy holds the promise not only of expanding current treatment options, but also of intervening in previously untackled causal disease mechanisms with little side effects. This scientific statement provides a comprehensive overview of the various modalities of gene therapy used to treat heart failure and some of its risk factors, and their application in the clinical setting. It discusses specifically the possibilities of gene therapy for hereditary heart diseases and (non)-genetic heart failure. Furthermore, it addresses safety and clinical trial design issues and challenges for future regulatory strategies.
Keywords:	Adeno-Associated Viral Vector, Gene Editing, Gene Replacement, Gene Silencing, Gene Therapy, Heart Failure
Source:	European Journal of Heart Failure
ISSN:	1388-9842
Publisher:	Wiley
Volume:	27
Number:	1
Page Range:	5-25
Date:	January 2025
Official Publication:	https://doi.org/10.1002/ejhf.3516
PubMed:	View item in PubMed

Repository Staff Only: item control page

Download Statistics

Download Statistics

Download Statistics

Downloads

Downloads per month over past year