Cardiac Gene Therapy: Methods and Protocols

Part I Overview

1. Updates on Cardiac Gene Therapy Research and Methods: Overview of Cardiac Gene Therapy

Francisco J. Romeo, Spyros A. Marvopoulos, and Kiyotake Ishikawa

Part II Gene Suppression, Editing, and Reprogramming

2. Tough Decoy-Mediated Cardiac Gene Suppression

Changwon Kho

3. Direct Reprogramming of Adult Human Cardiac Fibroblasts into Induced Cardiomyocytes using miRcombo

Camilla Paoletti, Carla Divieto, and Valeria Chiono

4. CRISPR/Cas9 Gene Editing of RYR2 in Human iPSC Derived Cardiomyocytes to Probe Ca²⁺ Signaling Aberrancies of CPVT Arrhythmogenesis

Naohiro Yamaguchi, Xiao-Hua Zhang, and Martin Morad

5. Enhancing Cardiomyocyte Transcription using In Vivo CRISPR/Cas9 Systems

Eric Schoger and Laura C. Zelarayn

6. AAV Mediated Somatic Gene Editing for Cardiac and Skeletal Muscle in a Large Animal Model

Tilman Ziegler, Tarik Bozoglu, and Christian Kupatt

Part III Cardiac Gene Therapy Vectors and Promotors

7. Optimization of Synthesis of Modified mRNA

Jimeen Yoo and Lior Zangi

8. Design and Production of Heart Chamber-Specific AAV9 Vectors

Alina S. Bilal, Donna J. Thuerauf, Erik A. Blackwood, and Christopher C. Glembotski

9. Generation of Atrial-Specific Construct using Sarcolipin Promoter Associated CRM4 Enhancer

Dongtak Jeong

Part IV Cardiac Gene Delivery Methods

10. Cardiac Targeted Adeno-Associated Virus Injection in Rats

Michael G. Katz, Yoav Hadas, Adam S. Vincek, Nataly Shtraizent, Eric Schadt, and Efrat Eliyahu

11. Cardiac Gene Delivery in Large Animal Models-Antegrade Techniques

Spyros A. Mavropoulos, Kelly P. Yamada, Tomoki Sakata, and Kiyotake Ishikawa

12. Locked Nucleic Acid AntimiR Therapy for the Heart

Sabine Samolovac and Rabea Hinkel

13. Cardiac Gene Delivery in Large Animal Models: Selective Retrograde Venous Injection

Philipp Schlegel and Philip W. J. Raake

14. Endocardial Gene Delivery using NOGA Catheter System

Satu Siimes, Niko Jrvelinen, Henna Korpela, and Seppo Yl-Herttuala

15. Surgical Methods for Cardiac Gene Delivery in Large Animals

Michael G. Katz, Yoav Hadas, Adam S. Vincek, Nataly Shtraizent, Hylton P. Gordon, Peter Pastuszko, Eric Schadt, and Efrat Eliyahu

16. Atrial Gene Painting in Large Animal Model of Atrial Fibrillation

Weilan Mo and J. Kevin Donahue

17. Stent-Based Gene Delivery for Coronary Disease

Ivan S. Alferiev, Michael Chorny, Robert L. Wilensky, Robert J. Levy, and Ilia Fishbein

18. Selective Anti-AAV Antibody Depletion by Hemapheresis and Immunoadsorption

Alejandro Orlowski and Thomas Weber

19. Ex Vivo Delivery of Viral Vectors by Organ Perfusion for Cardiac Transplantation Gene Therapy

Michelle Mendiola Pla, Amy Evans, Paul Lezberg, and Dawn E. Bowles

Part V Pulmonary Hypertension

20. Intra-Airway Gene Delivery for Pulmonary Hypertension in Rodent Models

Malik Bisserier, Olivier Boucherat, Sebastien Bonnet, and Lahouaria Hadri

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