Cardiac Gene Therapy: Methods and Protocols

by Kiyotake Ishikawa (Editor)

Overview

This detailed book provides methodological information on cardiac gene delivery, from classic to state-of-the-art technologies and techniques. Efficient, cardiac-specific, and safe vectors, as well as refined vector delivery methods, are key for successful cardiac gene transfer and eventually for improving patients’ outcomes. Newer vectors and more efficient vector delivery methods have the potential to dramatically improve gene transduction efficacy, while novel gene manipulation techniques enforce the therapeutic power and broaden disease targets. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory prools, and tips on troubleshooting and avoiding known pitfalls.

Authoritative and practical, Cardiac Gene Therapy: Methods and Prools serves as a valuable tool for molecular biologists and physiologists in the cardiology field conducting cardiac gene transfer research, which will ultimately lead to further advancements in the vital field.

Product Details

ISBN-13:	9781071627099
Publisher:	Springer US
Publication date:	09/01/2023
Series:	Methods in Molecular Biology , #2573
Edition description:	2nd ed. 2022
Pages:	347
Product dimensions:	7.01(w) x 10.00(h) x (d)

Table of Contents

Current Methods in Cardiac Gene Therapy: Overview.- Silencing Genes in the Heart.- Generation of Efficient miRNA Inhibitors Using Tough Decoy Constructs.- Efficient Genome Editing in Induced Pluripotent Stem Cells with Engineered Nucleases In Vitro.- Direct Cardiac Reprogramming as a Novel Therapeutic Strategy for Treatment of Myocardial Infarction.- Production and Characterization of Vectors Based on the Cardiotropic AAV Serotype 9.- Cell-Based Measurement of Neutralizing Antibodies Against Adeno-Associated Virus (AAV).- Synthesis of Modified mRNA for Myocardial Delivery.- Exosomes-Based Gene Therapy for MicroRNA Delivery.- Lipidoid mRNA Nanoparticles for Myocardial Delivery in Rodents.- Gene Transfer in Isolated Adult Cardiomyocytes.- Gene Transfer in Cardiomyocytes Derived from ES and iPS Cells.- Gene Transfer to Rodent Hearts In Vivo.- Ultrasound-Targeted Microbubble Destruction for Cardiac Gene Delivery.- A Needleless Liquid Jet Injection Delivery Approach for Cardiac Gene Therapy.- Cardiac Gene Delivery in Large Animal Models: Antegrade Techniques.- Direct Myocardial Injection of Vectors.- Selective Pressure-Regulate Retroinfusion for Gene Therapy Application in Ischemic Heart Disease.- Cardiac Gene Delivery Using Recirculating Devices.- Molecular Cardiac Surgery with Recirculating Delivery (MCARD): Procedure and Vector Transfer.- Gene Delivery for the Generation of Bioartificial Pacemaker.- Gene Therapy for Post-Infarction Ventricular Tachycardia.- MicroRNA Delivery Strategies to the Lung in a Model of Pulmonary Hypertension.- Inhaled Gene Transfer for Pulmonary Circulation.

Editorial Reviews

Reviewer: Omer Iqbal, MD, FACC, FESC (Loyola University Medical Center)
Description: Given the rapid advances in cardiac gene therapy, this second edition book provides 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. The previous edition was published in 2016.
Purpose: According to the book, its detailed and practical protocols will be valuable tools for researchers in cardiology to conduct cardiac gene therapy research. The authors hope that the step-by-step methodologies provided will undoubtedly lead to further advancement and successful clinical translation of cardiac gene therapy. These objectives are worthy and the book meets them.
Audience: The book is written for researchers in cardiology to conduct cardiac gene therapy research. In addition to these researchers, clinical cardiologists may find this book helpful. Given the comprehensive scope of the methods and protocols given, the book meets the needs of the intended audience. The author is a credible authority and a well-recognized expert in the field of cardiac gene therapy based on credentials and publications.
Features: The book is divided into five parts. Part I provides an overview and contains one chapter covering updates on cardiac gene therapy research and methods. Part II deals with gene suppression, editing, and reprogramming and is sub-divided into five chapters covering important topics such as tough decoy-mediated cardiac gene suppression, direct reprogramming of fibroblasts into induced cardiomyocytes through microRNAs, procedures to establish CRISPR/Cas9 gene edited hiPSC-derived cardiomyocytes, methods on how to enhance cardiomyocyte transcription using in vivo CRISPR/Cas 9 systems, CRISPRa in mouse cardiomyocytes, and AAV mediated in vivo gene editing in pigs. Part III deals with cardiac gene therapy vectors and promotors and is sub-divided into three chapters dealing with optimization of synthesis of modified mRNA (modRNA) — a safe and effective vector for gene-based therapy having been validated through COVID-19 vaccines, which used modRNA technology to translate spike proteins. Other chapters describe the AAV9 cardiac chamber-specific vectors and generation of atrial-specific construct. Part IV deals with cardiac gene delivery methods and is sub-divided into 10 chapters covering adeno-associated virus-based vectors, the most promising DNA delivery vehicle; different catheter-based antegrade gene delivery techniques; cardiac LNA therapy; selective retrograde venous injections; NOGA catheter delivery system for endocardial gene delivery; surgical gene delivery to the heart; and other stent-based gene delivery for coronary arteries. Part V deals with pulmonary hypertension and is sub-divided into five chapters covering important topics such as experimental techniques to determine the presence of neutralizing antibodies against AAV in the blood; protocol for obtaining carotid artery access and insertion of a pressure-volume catheter into the left ventricle of rodents; large animal P-V loop measurement; large animal ELISpot assay; and, lastly, methods for detection of shed AAV particles after cardiac gene therapy. The book has several colored illustrations, which make complex and difficult topics easy to understand. The index could have been expanded for locating topics of interest with greater ease.
Assessment: This book is excellent and will be highly useful for researchers in cardiology to conduct cardiac gene therapy research. The step-by-step methodology provided by experts in the field will surely lead to further advancements and successful clinical translation. Compared to other books on this topic, this book is unique as it provides updated protocols, many of them written by expert investigators with long-term expertise in cardiac gene therapy. The updated edition justifies replacement of the previous edition.

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