Cardiac gene delivery using ultrasound: State of the field

Singh, Davindra; Memari, Elahe; He, Stephanie; Yusefi, Hossein; Helfield, Brandon

doi:10.1016/j.omtm.2024.101277

Molecular Therapy - Methods & Clinical Development

2024

DOI: 10.1016/j.omtm.2024.101277

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Cardiac gene delivery using ultrasound: State of the field

Davindra Singh,

Elahe Memari,

Stephanie He

et al.

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Cited by 2 publications

References 154 publications

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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

Van Linthout,

Stellos,

Giacca

et al. 2024

European J of Heart Fail

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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.

show abstract

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

Van Linthout,

Stellos,

Giacca

et al. 2024

European J of Heart Fail

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The effect of micro-vessel viscosity on the resonance response of a two-microbubble system

Yusefi,

Helfield

2025

Ultrasonics

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Cardiac gene delivery using ultrasound: State of the field

Cited by 2 publications

References 154 publications

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

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

The effect of micro-vessel viscosity on the resonance response of a two-microbubble system

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