Gene therapy, including antisense oligonucleotides, siRNA, AAV vectors, and CRISPR-Cas9, holds promise for treating hereditary and non-genetic heart failure by targeting causal disease mechanisms.
This ESC scientific statement provides a comprehensive overview of current and emerging gene therapy strategies for heart failure, highlighting their potential to address causal disease mechanisms.
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.
Linthout et al. (Fri,) conducted a review in Heart failure. Gene therapy was evaluated. Gene therapy, including antisense oligonucleotides, siRNA, AAV vectors, and CRISPR-Cas9, holds promise for treating hereditary and non-genetic heart failure by targeting causal disease mechanisms.