This volume aims to explore the latest developments in adeno-associated viral and lentiviral vectors as well as the gene therapy strategies for the most common neurological disorders, followed by chapters that include step-by-step guides to viral vector-based gene delivery in animal models used in the authors' laboratories. Although safe gene manipulation in neural cells can be achieved, it may still be years away from efficacious gene-based treatment of neurological disorders such as Parkinson's and Alzheimer's diseases due to the complexity of the underlying genetic/molecular mechanisms and the difficulty of developing reliable animal models. Gene Delivery and Therapy for Neurological Disorders seeks to aid researchers in this vital work. Written in the popular Neuromethods series format, chapters include the kind of detailed description and expert implementation advice that leads to success in the lab. Meticulous and authoritative, Gene Delivery and Therapy for Neurological Disorders serves as an ideal guide for researchers attempting to explore the potentials of gene therapy for neurological disorders.

This book reviews the chemical, regulatory, and physiological mechanisms of protein arginine and lysine methyltransferases, as well as nucleic acid methylations and methylating enzymes. Protein and nucleic acid methylation play key and diverse roles in cellular signalling and regulating macromolecular cell functions. Protein arginine and lysine methyltransferases are the predominant enzymes that catalyse S-adenosylmethionine (SAM)-dependent methylation of protein substrates. These enzymes catalyse a nucleophilic substitution of a methyl group to an arginine or lysine side chain nitrogen (N) atom. Cells also have additional protein methyltransferases, which target other amino acids in peptidyl side chains or N-termini and C-termini, such as glutamate, glutamine, and histidine. All these protein methyltransferases use a similar mechanism. In contrast, nucleic acids (DNA and RNA) are substrates for methylating enzymes, which employ various chemical mechanisms to methylate nucleosides at nitrogen (N), oxygen (O), and carbon (C) atoms. This book illustrates how, thanks to there ability to expand their repertoire of functions to the modified substrates, protein and nucleic acid methylation processes play a key role in cells.

This open access book offers the first comprehensive account of the pan-genome concept and its manifold implications. The realization that the genetic repertoire of a biological species always encompasses more than the genome of each individual is one of the earliest examples of big data in biology that opened biology to the unbounded. The study of genetic variation observed within a species challenges existing views and has profound consequences for our understanding of the fundamental mechanisms underpinning bacterial biology and evolution. The underlying rationale extends well beyond the initial prokaryotic focus to all kingdoms of life and evolves into similar concepts for metagenomes, phenomes and epigenomes. The book's respective chapters address a range of topics, from the serendipitous emergence of the pan-genome concept and its impacts on the fields of microbiology, vaccinology and antimicrobial resistance, to the study of microbial communities, bioinformatic applications and mathematical models that tie in with complex systems and economic theory. Given its scope, the book will appeal to a broad readership interested in population dynamics, evolutionary biology and genomics.

RNA binding proteins are an exciting area of research in gene regulation. A multitude of RNA-protein interactions are used to regulate gene expression including pre-mRNA splicing, polyadenylation, editing, transport, cytoplasmic targeting, translation and mRNA turnover. In addition to these post-transcriptional processes, RNA-protein interactions play a key role in transcription as illustrated by the life cycle of retroviruses. Unlike DNA, the structure of RNA is highly variable and conformationally flexible, thus creating a number of unique binding sites and the potential for complex regulation by RNA binding proteins. Although there is a wide range of topics included in this volume, general themes have been repeated, highlighting the overall integrative nature of RNA binding proteins. The chapters have been separated into three different sections: Translational Control; mRNA Metabolism; and Hormonal and Homeostatic Regulation. The chapters of this volume were written with the seasoned investigator and student in mind. Summaries of key concepts are reviewed within each chapter as well as guiding questions that can be used to stimulate class discussions. The Editors of this volume hope that this compendium educates, enthralls, and stimulates the readers to look to the future possibilities in this rapidly evolving field.

This book contains a comprehensive series of reviews on the calcitonin gene-related peptide (CGRP) family of peptides. This family of peptide hormones has a diverse and constantly expanding range of important physiologic functions, including regulation of blood calcium, vascular tension, feeding behavior and pain recognition.

This volume includes chapters on:

• The adrenomedullin peptides and signalling
• Ligand binding and activation of the CGRP receptor
• Understanding amylin receptors
• The CGRP-receptor component protein
• The calcitonin peptide family
• Genetic regulation of CGRP
• Vascular actions of CGRP and adrenomedullin
• Intermedin/adrenomedullin 2 function
• CGRP and adrenomedullin as pain-related peptides
• Amylinergic control of ingestive behaviour
• Calcitonin receptors

This book discusses their receptors, physiological and pathophysiological functions and potential as clinical targets. It will appeal to researchers who study any of these peptides and those with an interest in migraine therapy due to the involvement of CGRP in this disorder. The book is unique because it brings together research on the whole peptide family for the first time in several years. It will be a useful reference volume for researchers in this area. This book will also appeal to researchers in the broader field of bioactive peptides.

About 7 million people worldwide are suffering from various inherited neuromuscular diseases. Gene therapy brings the hope of treating these diseases at their genetic roots. Muscle Gene Therapy is the only book dedicated to this topic. The first edition was published in 2010 when the field was just about to enter its prime time. The progress made since then has been unprecedented. The number of diseases that have been targeted by gene therapy has increased tremendously. The gene therapy toolbox is expanded greatly with many creative novel strategies (such as genome editing and therapy with disease-modifying genes). Most importantly, clinical benefits have begun to emerge in human patients. To reflect rapid advances in the field, we have compiled the second edition of Muscle Gene Therapy with contributions from experts that have conducted gene therapy studies either in animal models and/or in human patients. The new edition offers a much needed, up-to-date overview and perspective on the foundation and current status of neuromuscular disease gene therapy. It provides a framework to the development and regulatory approval of muscle gene therapy drugs in the upcoming years. This book is a must-have for anyone who is interested in neuromuscular disease gene therapy including those in the research arena (established investigators and trainees in the fields of clinical practice, veterinary medicine and basic biomedical sciences), funding and regulatory agencies, and patient community.

The aim of molecular diagnostics is preferentially to detect a developing disease before any symptoms appear. There has been a significant increase, fueled by technologies from the human genome project, in the availability of nucleic acid sequence information for all living organisms including bacteria and viruses. When combined with a different type of instrumentation applied, the resulting diagnostics is specific and sensitive. Nucleic acid-based medical diagnosis detects specific DNAs or RNAs from the infecting organism or virus and a specific gene or the expression of a gene associated with a disease. Nucleic acid approaches also stimulate a basic science by opening lines of inquiry that will lead to greater understanding of the molecules at the center of life. One can follow Richard Feynman's famous statement "What I cannot create, I do not understand."

Paris is a cosmopolitan city where roaring life, wonderful museums and excellent science can be found. It was during the XI IUMS conference held in this city that the Pseudomonas book series was ?rst envisaged. On the ?rst row of the auditorium sat a group of outstanding scientists in the ?eld, who after devoting much of their valuable time, contributed in an exceptional manner to the ?rst three volumes of the series, which saw the light simultaneously. The volumes were grouped under the generic titles of "Vol. I. Pseudomonas: Genomics, Life Style and Molecular Architecture", Vol. II. Pseudomonas: Virulence and gene regulation; Vol. III. Pseudomonas: Biosynthesis of Macromolecules and Molecular Metabolism. Soon after the completion of the ?rst three volumes, a rapid search for ar- cles containing the word Pseudomonas in the title in the last 10 years produced over 6,000 articles! Consequently, not all possible topics relevant to this genus were covered in the three ?rst volumes. Since then two other volumes were p- lished: Pseudomonas volume IV edited by Roger Levesque and Juan L. Ramos that came to being with the intention of collecting some of the most relevant emerging new issues that had not been dealt with in the three previous volumes. This v- ume was arranged after the Pseudomonas meeting organized by Roger Levesque in Quebec (Canada). It dealt with various topics grouped under a common heading: "Pseudomonas: Molecular Biology of Emerging Issues".

The purpose of this volume of Methods in Molecular Medicine is to set forth examples of the great variety of techniques and applications that are now emerging in the field of nonviral gene therapy. The book emphasizes not only specific approaches to gene delivery but, in particular, the best current me- ods to prepare, handle, and characterize gene delivery agents. These topics are of very broad importance since gene therapy evolves from its mostly ac- emy-based experimental and clinical research to the ever increasing number of industry-driven programs directed toward commercial development. S- cessful introduction of nonviral gene therapy agents into the clinic should be expected to require rigorous manufacturing and analytical methods that readily meet the regulatory guidelines under which new drug candidates are reviewed for marketing approval. Exactly what those guidelines will prove to be c- tainly depends on the established guidelines for review of both biological and chemical therapeutics. Additionally, many new techniques are being devised and applied to gene therapy research; these techniques will be instrumental in developing and characterizing successful gene delivery agents. Nonviral Vectors for Gene Therapy: Methods and Protocols has two main sections. To start with, there is a series of chapters on specific protocols for the synthesis, characterization, and application of gene delivery agents. S- eral chapters address the topic of materials to bind with DNA to form the compact condensed phases that facilitate cellular delivery.

This book presents descriptive overviews of gene editing strategies across multiple species while also offering in-depth insight on complex cases of application in the field of tissue engineering and regenerative medicine. Chapters feature contributions from leaders in stem cell therapy and biology, providing a comprehensive view of the application of gene therapy in numerous fields with an emphasis on ophthalmology, stem cells, and agriculture. The book also highlights recent major technological advances, including ZFN, TALEN, and CRISPR. Precision Medicine, CRISPR, and Genome Engineering is part of the highly successful Advances in Experimental Medicine and Biology series. It is an indispensable resource for researchers and students in genetics as well as clinicians.

This volume is a review which presents both a basic science and clinical perspective on neuroprotective approaches to acute and chronic neurodegenerative conditions. Experts from both fields review current areas of neuroprotection. The book describes basic science discovery in stroke research and the application of such research within the pharmaceutical industry leading to the development of neuroprotective drugs.

This volume details practical procedures on the latest DNA vaccine technology. Chapters guide readers through methods and protocols on DNA vaccine design, the adjuvant influence, production and purification methodologies, delivery systems, and approaches of the influence of DNA vaccines in the immunological response performance and in the cancer immunotherapy. 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. Authoritative and cutting-edge, DNA Vaccines: Methods and Protocols aims to ensure successful results in the further study of this vital field.

This third edition provides new and updated chapters on gene therapeutic strategies of cancer. Chapters guide readers through suicide and oncolytic gene therapy, gene replacement and gene suppression therapy, vector development and refinement, immunogene therapy, TCR and CAR engineering, tumor vaccination using DNA or RNA vaccines, and antitumoral immune stimulation at different levels. Written in the format of the highly successful Methods in Molecular Biology series, each chapter includes an introduction to the topic, lists necessary materials and reagents, includes tips on troubleshooting and known pitfalls, and step-by-step, readily reproducible protocols. Authoritative and cutting-edge, Gene Therapy of Cancer: Methods and Protocols, Third Edition aims to be a useful and practical guide to new researchers and experts looking to expand their knowledge.

Sixteen contributions cover such topics as the polymerase chain reaction; regulation of alternative splicing; human retinoblastoma susceptibility gene; control of translation initiation in mammalian cells; the utility of streptomycetes as hosts for gene cloning; folding of eukaryotic proteins produc

This book is a collection of principles and current practices in omics research, applied to skeletal muscle physiology and disorders. The various sections are categorized according to the level of biological organization, namely, genomics (DNA), transcriptomics (RNA), proteomics (protein), and metabolomics (metabolite). With skeletal muscle as the unifying theme, and featuring contributions from leading experts in this traditional field of research, it highlights the importance of skeletal muscle tissue in human development, health and successful ageing. It also discusses other fascinating topics like developmental biology, muscular dystrophies, exercise, insulin resistance and atrophy due to disuse, ageing or other muscle diseases, conveying the vast opportunities for generating new hypotheses as well as testing existing hypotheses by combining high-throughput techniques with proper experiment designs, bioinformatics and statistical analyses. Presenting the latest research techniques, this book is a valuable resource for the physiology community, particularly researchers and grad students who want to explore the new opportunities for omics technologies in basic physiology research.

This book examines the basic cellular and molecular mechanisms associated with aging. It comprehensively describes the genetic, epigenetic, biochemical and metabolic regulation of aging, as well as some important age-related diseases. Divided into two major sections, it takes readers through the various aspects of aging in a story-like manner and suggests various interventions for healthy aging, such as dietary restriction, regular exercise, nutrition and maintaining a balanced and a non-stressful lifestyle. It describes the implications of aging on the nervous system, metabolism, immunity and stem cells as well as care for the elderly. The book is an ideal companion for both new and established researchers in the field and is also useful for educators, clinicians and policy makers.

GW bodies are novel cytoplasmic foci that were discovered and named by Dr. Chan's group in 2002. These bodies are now known to be active cytoplasmic foci involved with the new gene regulation process mediated by microRNA that leads to translational repression and mRNA degradation. The detailed biological functions of these cytoplasmic structures are still being uncovered and the idea for this book is to provide the history of the discovery and the major work from different laboratories that has led to the characterization and elucidation of the structure and function of these new multiple subcellular structures.

This detailed volume assembles a number of the most commonly used and state-of-the-art methodologies in the revolutionary field of mRNA processing in order to aid researchers in choosing the best method for their specific problems. Given that the vast majority of alternative mRNA processing events has yet to be explored, these techniques promise to breathe new life into the myriad new ideas currently underway. 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. Practical and easy to use, mRNA Processing: Methods and Protocols seeks to aid scientists venturing into what's certain to be another explosive period of growth in this dynamic field of study. The chapter 'Robust, Cost-Effective Profiling of RNA Binding Protein Targets with Single-end Enhanced Crosslinking and Immunoprecipitation (seCLIP)' is published open access under a CC BY 4.0 license at link.springer.com.

In a simplified form, epigenetics refers to heritable changes in phenotype that are not due to changes in the underlying DNA sequence. In this book, epigenetic mechanisms of regulation and dysregulation in health and disease are explored in great depth. Detailed chapters on epigenetic processes including DNA methylation and chromatin post-translational modifications including potential interventions with DNA methyltransferase inhibitors and histone deacetylase inhibitors are explored in initial chapters. These provide a detailed overview and important background to the entire field. The book is then focussed on epigenetic mechanisms involved in various diseases including anti-inflammatory and autoimmune conditions. Important accounts relating to the effects of epigenetics in metabolic syndrome, cardiovascular disease and asthma are the focus of subsequent chapters. The role of epigenetic dysregulation in malignancy is a current topic of interest and represents an intense field of research. A large component of this book is dedicated to the analysis of aberrant epigenetic processes in carcinogenesis and cancer progression. Further, chapters are focused on emerging cancer prevention using nutritional components and anti-cancer therapies particularly with histone deacetylase inhibitors, which have already been approved for the treatment of cutaneous T-cell lymphoma. The emerging role of nanoparticle preparations, especially in the context of delivering potential epigenetic therapies to target cells in various diseases, is also explored in this book. Overall, this book encompasses a wide range of topics related to epigenetic mechanisms in health and disease and would appeal to anyone with an interest in epigenetics, chromatin biology and emerging epigenetic interventions and therapies.

This volume gathers a selection of original articles and reviews on timely topics about the application of Taurine in human health written by members of the International Taurine Society, including COVID-19, cancer, heart disease, and diabetes, among others. Chapters are written by Taurine experts across the globe in North and South America, Asia, and Europe. A majority of the articles are based on original studies recently carried out in individual laboratories worldwide. The book is divided into eight parts, each covering a unique aspect of Taurine. Each section will highlight new research findings on Taurine and its application in various human systems, including the nervous system, immune system, and cardiovascular system, to combat disease. The first section covers COVID-19, the dominant health event of 2020. Experts will explore and clarify the potential therapeutic effectiveness of Taurine against COVID-19. The volume will promote further research into the application of Taurine in human health, and will be of use to a wide audience, including basic and clinical scientists, pharmaceutical and nutraceutical companies, and libraries.

Transcription Factor Regulatory Methods details various techniques ranging from cutting-edge to general techniques use to study transcription factor regulatory networks. 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 key tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Transcription Factor Regulatory Methods aids scientists in the further study into post-genomic or the personal genomic era.

This second edition volume brings together the experiences of leading scientists in the discipline of cancer gene profiling. Because cancer genes can be profiled in many different ways, Cancer Gene Profiling: Methods and Protocols, Second Edition explores different techniques and approaches needed to understand the key stages of cancer development, as using only one technique would be insufficient. This book provides readers with an overview of the state-of-the-art methods that will enable them to perform these experiments, and demonstrates the kind of analysis that is possible in our current biomedical research. 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. Thorough and cutting-edge, Cancer Gene Profiling: Methods and Protocols, Second Edition is a great resource for any student or practitioner with an interest in cancer gene profiling, and can be used in any well-equipped research laboratory.

In this book, leading experts provide timely and comprehensive information on methods for conditional mutagenesis in the mouse and their application to model human physiology and pathophysiology. The book illustrates how sophisticated genetic manipulations of the mouse genome are employed to model human diseases and to identify underlying molecular mechanisms. In addition, it considers the development of new drugs to treat human diseases.

This book provides an in depth review of our current knowledge on the role of telomeres and telomerase in ageing, disease and cancer. Telomere shortening results in telomere dysfunction, which represents one of the basic aspects of ageing contributing to the accumulation of DNA damage during ageing. The book describes cell intrinsic checkpoints (senescence and apoptosis) and environmental alterations that limit stem cell function during ageing and disease. The book focuses on experimental data in mouse models and cell lines and, in addition, provides and overview on telomere shortening in human diseases and ageing. The book also describes future directions in research on adult stem cell ageing, telomeres, and cancer. In addition, the book points to potential targets for molecular therapies aiming to improve regeneration and stem cell function during ageing or to impair cancer cell proliferation.

A special focus of the book is on adult stem cells. There is emerging evidence that adult stem cell ageing impairs organismal fitness and survival and contributes to cancer formation (cancer stem cells). The book summarizes basic mechanisms of adult stem cell ageing. Moreover, the authors describe consequences of telomere dysfunction on stem cell function involving cell intrinsic checkpoints as well as environmental alteration of the stem cell niche.

The above subjects appear to be of utmost importance to ageing researchers and physicians. Due to the advances in medicine, food supply and housing a growing percentage of humans reaches a long lifespan (>70 years). At this age molecular mechanisms of ageing limit organ maintenance, fitness and survival. A detailed understanding of the molecular mechanisms underlying the ageing process is essential to further improve quality of life and health span in the ageing human populations.

In summary, this book provides an excellent basis for academic teachers, scientists, and students interested in the areas of stem cell ageing, telomeres & telomerase, regeneration, and cancer. In addition, the book is of interest for physicians since the development of molecular therapies targeting ageing stem cells represents a promising approach to improve regenerative reserve and organ function during ageing and could also serve to target cancer stem cells.