Main description:

This second edition volume expands on the previous edition with updated techniques and discussions on topics such as gene suppression, editing, and reprogramming; cardiac gene therapy vectors and promoters; cardiac gene delivery methods; pulmonary hypertension; and patient screening and measuring the efficacy of cardiac gene therapy. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls.

Cutting-edge and comprehensive, Cardiac Gene Therapy: Methods and Protocols, Second Edition is a valuable tool for researchers in cardiology who are conducting gene therapy research. This book will lead to further advancements and successful clinical translations in the field.
Chapter "Direct Reprogramming of Adult Human Cardiac Fibroblasts into Induced Cardiomyocytes Using miRcombo" is available open access under a Creative Commons Attribution 4.0 International License via link.springer.com.

Contents:

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. Zelarayan

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 Jarvelainen, Henna Korpela, and Seppo Yla-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

21. Endo-Bronchial Gene Delivery for Pulmonary Hypertension in a Large Animal Model

Olympia Bikou and Kiyotake Ishikawa

Part VI Patient Screening and Measuring the Efficacy of Cardiac Gene Therapy

22. Cell-Based Determination of Neutralizing Antibodies against Adeno-Associated Virus in Cardiac Gene Therapy

Anjali J. Ravichandran, Renata Mazurek, and Kiyotake Ishikawa

23. Left Ventricular Pressure Volume Assessment Using Carotid Artery Access in the Rat

Spyros A. Mavropoulos and Kiyotake Ishikawa

24. Assessing the Effect of Cardiac Gene Therapy using Catheter-Based Pressure-Volume Measurement in Large Animals

Tomoki Sakata, Renata Mazurek, Spyros A. Mavropoulos, Francisco J. Romeo, Anjali J. Ravichandran, and Kiyotake Ishikawa

25. ELIspot Assay for Gene Therapy in Large Animal Studies

Renata Mazurek and Kiyotake Ishikawa

26. Assessing Recombinant AAV Shedding after Cardiac Gene Therapy

Melad Farraha and Eddy Kizana