TY - JOUR
T1 - State of the art and perspectives of gene therapy in heart failure
T2 - 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
AU - Van Linthout, Sophie
AU - Stellos, Konstantinos
AU - Giacca, Mauro
AU - Bertero, Edoardo
AU - Cannata, Antonio
AU - Carrier, Lucie
AU - Garcia-Pavia, Pablo
AU - Ghigo, Alessandra
AU - González, Arantxa
AU - Haugaa, Kristina H.
AU - Imazio, Massimo
AU - Lopes, Luis R.
AU - Most, Patrick
AU - Pollesello, Piero
AU - Schunkert, Heribert
AU - Streckfuss-Bömeke, Katrin
AU - Thum, Thomas
AU - Tocchetti, Carlo Gabriele
AU - Tschöpe, Carsten
AU - van der Meer, Peter
AU - van Rooij, Eva
AU - Metra, Marco
AU - Rosano, Giuseppe M.C.
AU - Heymans, Stephane
N1 - Publisher Copyright:
© 2024 The Author(s). European Journal of Heart Failure published by John Wiley & Sons Ltd on behalf of European Society of Cardiology.
PY - 2025/1
Y1 - 2025/1
N2 - 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.
AB - 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.
KW - Adeno-associated viral vector
KW - Gene editing
KW - Gene replacement
KW - Gene silencing
KW - Gene therapy
KW - Heart failure
UR - http://www.scopus.com/inward/record.url?scp=85210042381&partnerID=8YFLogxK
U2 - 10.1002/ejhf.3516
DO - 10.1002/ejhf.3516
M3 - Article
AN - SCOPUS:85210042381
SN - 1388-9842
VL - 27
SP - 5
EP - 25
JO - European Journal of Heart Failure
JF - European Journal of Heart Failure
IS - 1
ER -