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Gene and Cell Therapy

Gene and Cell Therapy Author Nancy Smyth Templeton
ISBN-10 9781466572003
Release 2015-01-20
Pages 1337
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The Most Comprehensive, State-of-the-Art Book on Using Gene and Cell Therapy in Clinical Medicine Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, Fourth Edition presents extensive background and basic information, state-of-the-art technologies, important achievements, and lingering challenges in the fields of gene and cell therapies. The fourth edition of this bestseller continues to provide the most comprehensive coverage of these fields in one volume. Some chapters have expanded introductions, making the book even more suitable for classroom use. This edition also offers more material on the contributors’ research efforts as well as current technologies, disease targets, and clinical applications. Divided into four sections, the book covers: Delivery systems and therapeutic strategies Other therapeutic strategies, including technologies that knock down gene expression Gene expression, regulation, and detection Gene and cell therapies, disease targets, clinical trials, and regulatory issues Accessible to a broad audience, including students, scientists, physicians, and lay people, this book provides readers with up-to-date, interdisciplinary knowledge and tools to tackle the evolving areas of gene therapy, cell therapy, and tissue engineering. From introductory information to state-of-the-art technologies and concepts, the book helps readers understand vector design and construction, delivery systems, therapeutic strategies, gene expression and detection, disease targets, clinical applications and trials, cell-based therapies, novel imaging systems, gene regulation, and regulatory affairs.

Gene Therapy

Gene Therapy Author Nancy Smyth Templeton
ISBN-10 UOM:39015050321036
Release 2000-06-15
Pages 584
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This text covers all aspects of gene therapy, including basic principles, viral and nonviral delivery systems, targeted diseases, regulatory issues, and FDA requirements. It investigates genetic bullets to block HIV-1 replication and genetic guns to deliver antiviral agents.

Advanced Textbook on Gene Transfer Gene Therapy and Genetic Pharmacology

Advanced Textbook on Gene Transfer  Gene Therapy and Genetic Pharmacology Author Daniel Scherman
ISBN-10 9781783263141
Release 2013-12-10
Pages 556
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This unique advanced textbook provides a clear and comprehensive description of the field of gene delivery, gene therapy and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations. The use of gene transfer is exponentially growing in the scientific and medical communities for day-to-day cell biology experiments and swift development of revolutionary gene therapy strategies. In this advanced textbook, more than 25 leading scientists, world-renowned in their respective fields, come together to provide a clear and comprehensive description of gene delivery, gene therapy and genetic pharmacology. This educational introduction to the main gene transfer vectors and selected therapeutic applications provides the background material needed to further explore the subject as well as relevant research literature. It will thus be invaluable to Master, PhD or MD students, post-doctoral scientists or medical doctors, as well as any scientist wishing to deliver a gene or synthetic nucleotide, or develop a gene therapy strategy. Furthermore, the textbook's simple and synthetic content will be of value to any reader interested in the biological and medical revolution derived from the elucidation of the human genome. Contents:Basic Definitions and Principles:Introduction (Thierry VandenDriessche)Basic Definitions and General Principles (Daniel Scherman)History of Gene Therapy (Serge Braun)Genetic Pharmacology Using Synthetic Deoxyribonucleotides (Jean-Christophe François and Carine Giovannangeli)Principles of RNAi Trigger Expression for Gene Therapy (Lisa J Scherer and John J Rossi)On Demand Alternative Splicing for Gene Rescue (Stéphanie Lorain and Luis Garcia)Nuclease-Mediated Targeted Genetic Correction (Dieter C Gruenert, Hamid Emamekhoo and R Geoffrey Sargent)Vectors and Gene Delivery Techniques:γ-Retrovirus- and Lentivirus-Derived Vectors for Gene Transfer and Therapy (Caroline Duros and Odile Cohen-Haguenauer)Adenovirus Vectors (Stefan Kochanek)Adeno-Associated Virus (AAV) Vectors (Aurélie Ploquin, Hildegard Büning and Anna Salvetti)Herpes Simplex Virus (HSV-1)-Based Vectors: Applications for Gene Transfer, Gene Therapy, Cancer Virotherapy and Vaccination (Matias E Melendez, Aldo Pourchet, Anna Greco and Alberto L Epstein)Non-Viral DNA Vectors (Martin Schleef)Macromolecular Conjugates for Non-Viral Nucleic Acid Delivery (Mark Ericson, Kevin Rice and Guy Zuber)Auto-Associative Lipid-Based Systems for Non-Viral Nucleic Acid Delivery (Virginie Escriou, Nathalie Mignet and Andrew Miller)Hydrodynamic-Pressure-Based Non-Viral Nucleic Acid Delivery (Takeshi Suda, Kenya Kamimura, Guisheng Zhang and Dexi Liu)Electrotransfer/Electroporation for Non-Viral Nucleic Acid Delivery (Pascal Bigey, Richard Heller and Daniel Scherman)Imaging of Gene Delivery (Georges Vassaux, Peggy Richard-Fiardo, Béatrice Cambien and Philippe Franken)Therapeutic Applications:Oncolytic Adenoviruses for Cancer Gene Therapy (Gunnel Hallden, Yaohe Wang, Han-Hsi Wong and Nick R Lemoine)Progress in DNA Vaccine Approaches for Cancer Immunotherapy (Geoffrey D Hannigan and David B Weiner)Gene Therapy of the Beta-Hemoglobinopathies (Emmanuel Payen, Charlotte Colomb, Olivier Negre, Marina Cavazzana-Calvo, Salima Hacein-Bey-Abina, Yves Beuzard and Philippe Leboulch)Gene Therapy for Hemophilia A and B (Nisha Nair, Marinee Chuah and Thierry VandenDriessche)Experimental and Clinical Ocular Gene Therapy (Alexis-Pierre Bemelmans and José-Alain Sahel)Gene Therapy of Neurological Diseases (Lisa M Stanek, Lamya S Shihabuddin and Seng H Cheng)Genetic Therapy of Muscle Diseases: Duchenne Muscular Dystrophy (Takis Athanasopoulos, Susan Jarmin, Helen Foster, Keith Foster, Jagjeet Kang, Taeyoung Koo, Alberto Malerba, Linda Popplewell, Daniel Scherman and George Dickson)Induced Pluripotent Stem Cells and Gene Targeting for Regenerative Medicine (Jizhong Zou and Linzhao Cheng)Gene Vector Production:Production and Purification of Viral Vectors and Safety Considerations Related to Their Use (Otto-Wilhelm Merten, Matthias Schweizer, Parminder Chahal and Amine Kamen)Production and Purification of Plasmid Vectors (Martin Schleef) Readership: Master, PhD or MD students, post-doctoral scientists or medical doctors, and any scientists using gene transfer techniques or implementing gene therapy strategies. Key Features:The advanced textbook addresses a broader audience of Master degree students than existing literature dedicated to more specialized scientistsThe advanced textbook might be purchased by non-specialists, such as MDs who want to follow the evolution of modern medicineThe global field of nucleotide-based therapies and nucleotide delivery are presented in this comprehensive textbook in simple words, giving easy access to key concepts and technologiesThe textbook also aims at helping non-specialized scientists choose an adequate gene transfer and/or gene therapy strategyKeywords:Gene Transfer;Gene Therapy;Gene Delivery;Genetic Pharmacology;Genetic Diseases;Vectors;Nucleotide;Therapy

A Guide to Human Gene Therapy

A Guide to Human Gene Therapy Author Roland W. Herzog
ISBN-10 9789814280914
Release 2010
Pages 388
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Ever since the birth of molecular biology, the tantalizing possibility of treating disease at its genetic roots has become increasingly feasible. Gene therapy - though still in its infancy - remains one of the hottest areas of research in medicine. Its approach utilizes a gene transfer vehicle ( vector) to deliver therapeutic DNA or RNA to cells of the body in order to rectify the defect that is causing the disease. Successful therapies have been reported in humans in recent years such as cures in boys with severe immune deficiencies. Moreover, gene therapy strategies are being adapted in numerous biomedical laboratories to obtain novel treatments for a variety of diseases and to study basic biological aspects of disease. Correction of disease in animal studies, is steadily gaining ground, highlighting the immense potential of gene therapy in the medical profession. This book will cover topics that are at the forefront of biomedical research such as RNA interference, viral and non-viral gene transfer systems, treatment of hematological diseases and disorders of the central nervous system.Leading experts on the respective vector or disease will contribute the individual chapters and explain cutting-edge technologies. It also gives a broad overview of the most important gene transfer vectors and most extensively studied target diseases. This comprehensive guide is therefore a must-read for anyone in the biotechnology, biomedical or medical industries seeking to further their knowledge in the area of human gene therapy.

Adenoviral Vectors for Gene Therapy

Adenoviral Vectors for Gene Therapy Author David T. Curiel
ISBN-10 9780128005101
Release 2016-03-10
Pages 868
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Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement Demonstrates noninvasive imaging of adenovirus-mediated gene transfer Discusses utility of adenoviral vectors in animal disease models Considers Federal Drug Administration regulations for human clinical trials

Cellular Therapy for Neurological Injury

Cellular Therapy for Neurological Injury Author Charles S., Jr. Cox
ISBN-10 9781482225938
Release 2016-04-06
Pages 288
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Cellular Therapy for Neurological Injury discusses the current status of cellular therapy for neurological disorders. The primary areas of focus include traumatic brain injury, stroke (ischemic and hemorrhagic), and spinal cord injury. The book explores cell therapy approaches to these and other conditions, while discussing current advances and a literature review in the context of a field that is moving rapidly. The book presents a translational focus, addressing barriers and opportunities to moving forward. The mechanisms of injury are explored, as well as how these mechanisms influence the type of cell therapy used, the route of delivery, and dosing regimen. Written by leaders in the field, this is an invaluable resource for clinicians and researchers alike.

Principles of Tissue Engineering

Principles of Tissue Engineering Author Robert Lanza
ISBN-10 9780123983701
Release 2013-10-17
Pages 1936
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Now in its fourth edition, Principles of Tissue Engineering has been the definite resource in the field of tissue engineering for more than a decade. The fourth edition provides an update on this rapidly progressing field, combining the prerequisites for a general understanding of tissue growth and development, the tools and theoretical information needed to design tissues and organs, as well as a presentation by the world’s experts of what is currently known about each specific organ system. As in previous editions, this book creates a comprehensive work that strikes a balance among the diversity of subjects that are related to tissue engineering, including biology, chemistry, material science, and engineering, among others, while also emphasizing those research areas that are likely to be of clinical value in the future. This edition includes greatly expanded focus on stem cells, including induced pluripotent stem (iPS) cells, stem cell niches, and blood components from stem cells. This research has already produced applications in disease modeling, toxicity testing, drug development, and clinical therapies. This up-to-date coverage of stem cell biology and other emerging technologies –such as brain-machine interfaces for controlling bionics and neuroprostheses– is complemented by a series of new and updated chapters on recent clinical experience in applying tissue engineering, as well as a new section on the application of tissue-engineering techniques for food production. The result is a comprehensive textbook that will be useful to students and experts alike. Includes new chapters on biomaterial-protein interactions, nanocomposite and three-dimensional scaffolds, skin substitutes, spinal cord, vision enhancement, and heart valves Offers expanded coverage of adult and embryonic stem cells of the cardiovascular, hematopoietic, musculoskeletal, nervous, and other organ systems Full-color presentation throughout

Stem Cell and Gene Therapy for Cardiovascular Disease

Stem Cell and Gene Therapy for Cardiovascular Disease Author Emerson C. Perin
ISBN-10 9780128018637
Release 2015-08-21
Pages 538
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Stem Cell and Gene Therapy for Cardiovascular Disease is a state-of-the-art reference that combines, in one place, the breadth and depth of information available on the topic. As stem cell and gene therapies are the most cutting-edge therapies currently available for patients with heart failure, each section of the book provides information on medical trials from contributors and specialists from around the world, including not only what has been completed, but also what is planned for future research and trials. Cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals will find this book an invaluable resource for further study on the topic. Provides information on stem and gene therapy medical trials from contributors and specialists around the world, including not only what has been completed, but also what is planned for future research and trials Presents topics that can be applied to allogeneic cells, mesenchymal cells, gene therapy, cardiomyoctyes, iPS cells, MAPC's, and organogenesis Covers the three areas with the greatest clinical trials to date: chronic limb ischemia, chronic angina, and acute MI Covers the prevailing opinions on how to harness the body’s natural repair mechanisms Ideal resource for cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals

Molecular Biology of Cancer

Molecular Biology of Cancer Author Lauren Pecorino
ISBN-10 9780199577170
Release 2012-04-26
Pages 342
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The third edition of The Molecular Biology of Cancer: Mechanisms, Targets, and Therapeutics offers a fresh approach to the study of the molecular basis of cancer, by showing how our understanding of the defective mechanisms which drive cancer is leading to the development of new targeted therapeutic agents.

A Concise Introduction to Pure Mathematics Fourth Edition

A Concise Introduction to Pure Mathematics  Fourth Edition Author Martin Liebeck
ISBN-10 9781498722933
Release 2015-10-28
Pages 301
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Accessible to all students with a sound background in high school mathematics, A Concise Introduction to Pure Mathematics, Fourth Edition presents some of the most fundamental and beautiful ideas in pure mathematics. It covers not only standard material but also many interesting topics not usually encountered at this level, such as the theory of solving cubic equations; Euler’s formula for the numbers of corners, edges, and faces of a solid object and the five Platonic solids; the use of prime numbers to encode and decode secret information; the theory of how to compare the sizes of two infinite sets; and the rigorous theory of limits and continuous functions. New to the Fourth Edition Two new chapters that serve as an introduction to abstract algebra via the theory of groups, covering abstract reasoning as well as many examples and applications New material on inequalities, counting methods, the inclusion-exclusion principle, and Euler’s phi function Numerous new exercises, with solutions to the odd-numbered ones Through careful explanations and examples, this popular textbook illustrates the power and beauty of basic mathematical concepts in number theory, discrete mathematics, analysis, and abstract algebra. Written in a rigorous yet accessible style, it continues to provide a robust bridge between high school and higher-level mathematics, enabling students to study more advanced courses in abstract algebra and analysis.

Therapeutic Applications of Adenoviruses

Therapeutic Applications of Adenoviruses Author Philip Ng
ISBN-10 9781498745499
Release 2016-12-12
Pages 256
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Adenoviruses are double stranded DNA viruses that have been used to study the process of DNA replication. Studies of the mode of action of adenovirally produced tumors in rodents led to the discovery of tumour supressor genes. The adenoviral vector is now the most used vector in clinical gene therapy especially for some kinds of cancers. The chapters in this book focus on the most up-to-date developments in the therapeutic applications of adenoviruses. The intended audience is individuals in the Life Sciences interested in therapeutic applications of adenoviruses. This book reviews the life history and immune responses to adenoviruses and summarizes various therapies implemented with the use of adenoviruses.

Spinal Muscular Atrophy

Spinal Muscular Atrophy Author Charlotte J Sumner
ISBN-10 9780128036860
Release 2016-10-24
Pages 506
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Spinal Muscular Atrophy: Disease Mechanisms and Therapy provides the latest information on a condition that is characterized by motoneuron loss and muscle atrophy, and is the leading genetic cause of infant mortality. Since the identification of the gene responsible for SMA in 1995, there have been important advances in the basic understanding of disease mechanisms, and in therapeutic development. This book provides a comprehensive accounting of recent advances in basic and clinical research that covers SMA clinical features and standards of care, multifaceted aspects of SMN protein functions and SMA disease pathology, various animal models, and biomarkers, as well as current therapeutic development. This title is ideal for graduate students/postdocs and principal investigators who are already in the SMA field and need to keep updated on recent findings and approaches, and for those who are new to, or would like to join, the field. Likewise, users will find an excellent source of reading for biotech/pharma scientists, clinical researchers, and practitioners, regulators, and patients and their advocacy organizations. Furthermore, this book is a handy reference for researchers and clinicians who may want to apply the research strategies and therapeutic approaches in SMA to other rare diseases. Provides comprehensive, up-to-date reviews by leading investigators on diverse topics of SMA, including clinical features and patient care, SMN genetics and protein functions, animal models, disease pathology and mechanisms, biomarkers, current therapeutic development, and the role of non-profit organizations in therapeutic development Written to bridge multiple disciplines and promote better communications among basic scientists, clinical researchers, and health care providers on the latest developments in SMA Includes outstanding questions and perspectives for future investigations and key references for additional detailed study

Multifunctional Nanocarriers for Contemporary Healthcare Applications

Multifunctional Nanocarriers for Contemporary Healthcare Applications Author Barkat, Md. Abul
ISBN-10 9781522547822
Release 2018-01-12
Pages 602
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Advances in technology permeates every aspect of life, including the healthcare system. Nanotechnology based systems have gained popularity based upon their promise, size, and other characteristics. Multifunctional Nanocarriers for Contemporary Healthcare Applications is a critical academic publication that explores advancements in nanostructured systems, applications of these systems in healthcare, and biomedical applications of these systems. Featuring coverage on a wide range of topics, such as hydrogels, controlled drug delivery systems, and nanomedicine, this book is geared toward researchers, students, and academicians seeking current research on advancements and applications of nanostructured systems in the healthcare industry.

Jasper s Basic Mechanisms of the Epilepsies

Jasper s Basic Mechanisms of the Epilepsies Author Jeffrey Noebels
ISBN-10 9780199842599
Release 2012-06-01
Pages 1264
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H.H. Jasper, A.A. Ward, A. Pope and H.H. Merritt, chair of the Public Health Service Advisory Committee on the Epilepsies, National Institutes of Health, published the first volume on Basic Mechanisms of the Epilepsies (BME) in 1969. Their ultimate goal was to search for a "better understanding of the epilepsies and seek more rational methods of their prevention and treatment." Since then, basic and clinical researchers in epilepsy have gathered together every decade and a half with these goals in mind -- assessing where epilepsy research has been, what it has accomplished, and where it should go. In 1999, the third volume of BME was named in honor of H.H. Jasper. In line with the enormous expansion in the understanding of basic epilepsy mechanisms over the past four decades, this fourth edition of Jasper's BME is the most ambitious yet. In 90 chapters, the book considers the role of interactions between neurons, synapses, and glia in the initiation, spread and arrest of seizures. It examines mechanisms of excitability, synchronization, seizure susceptibility, and ultimately epileptogenesis. It provides a framework for expanding the epilepsy genome and understanding the complex heredity responsible for common epilepsies as it explores disease mechanisms of ion channelopathies and developmental epilepsy genes. It considers the mechanisms of conditions of epilepsy comorbidities. And, for the first time, this 4th edition describes the current efforts to translate the discoveries in epilepsy disease mechanisms into new therapeutic strategies. This book, considered the 'bible' of basic epilepsy research, is essential for the student, the clinician scientist and all research scientists who conduct laboratory-based experimental epilepsy research using cellular, brain slice and animal models, as well as for those interested in related disciplines of neuronal oscillations, network plasticity, and signaling in brain strucutres that include the cortex, hippocampus, and thalamus. In keeping with the 1969 goals, the book is now of practical importance to the clinical neurologist and epileptologist as the progress of research in molecular genetics and modern efforts to design antiepileptic drugs, cures and repairs in the epilepsies converge and impact clinical care.

Molecular Biology of the Cell

Molecular Biology of the Cell Author Bruce Alberts
ISBN-10 9781317563747
Release 2017-08-07
Pages 1464
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As the amount of information in biology expands dramatically, it becomes increasingly important for textbooks to distill the vast amount of scientific knowledge into concise principles and enduring concepts.As with previous editions, Molecular Biology of the Cell, Sixth Edition accomplishes this goal with clear writing and beautiful illustrations. The Sixth Edition has been extensively revised and updated with the latest research in the field of cell biology, and it provides an exceptional framework for teaching and learning. The entire illustration program has been greatly enhanced.Protein structures better illustrate structure–function relationships, icons are simpler and more consistent within and between chapters, and micrographs have been refreshed and updated with newer, clearer, or better images. As a new feature, each chapter now contains intriguing openended questions highlighting “What We Don’t Know,” introducing students to challenging areas of future research. Updated end-of-chapter problems reflect new research discussed in the text, and these problems have been expanded to all chapters by adding questions on developmental biology, tissues and stem cells, pathogens, and the immune system.


Placenta Author Ornella Parolini
ISBN-10 9781498700276
Release 2016-02-22
Pages 296
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Long regarded as biological waste, the placenta is gaining momentum as a viable product for clinical use. Due to their unique properties, placental cells and derivatives show great promise in curing various diseases. Utilizing contributions from world-renowned experts, Placenta: The Tree of Life considers the therapeutic potential of these cells. It examines new stem cell–based strategies and highlights recent studies that advance the range of treatment for a number of illnesses. Emphasizing the potential research and therapeutic use of stem cells, the book discusses the development, structure, and functions of the human placenta. It introduces overall aspects of the immune system, explains some of the immune mechanisms during pregnancy, and shows the role of the placenta in these mechanisms. Current scientific research is presented that focuses on the mechanisms of action underlying the therapeutic benefit of cells isolated from different placental regions. An exhaustive examination, this pivotal work: Considers how perinatal cells may represent an important source for cell therapy approaches in the near future, in both human and veterinary medicine Describes the clinical potential of placenta-derived cells in regenerative medicine—specifically in neurological disorders, metabolic liver diseases, inflammatory diseases, and autoimmune diseases Explains how cells isolated from different placental tissues share basic properties Placenta: The Tree of Life summarizes the advantages of perinatal tissue as a source of cells with therapeutic potential and is designed for use in the study of genetics, stem cell science, placental function, reproductive biology, regenerative medicine, and related fields.

Designing Clinical Research

Designing Clinical Research Author Stephen B. Hulley
ISBN-10 9781469840543
Release 2013-05-08
Pages 378
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Designing Clinical Research sets the standard for providing a practical guide to planning, tabulating, formulating, and implementing clinical research, with an easy-to-read, uncomplicated presentation. This product incorporates current research methodology--including molecular and genetic clinical research--and offers an updated syllabus for conducting a clinical research workshop. Emphasis is on common sense as the main ingredient of good science. The book explains how to choose well-focused research questions and details the steps through all the elements of study design, data collection, quality assurance, and basic grant-writing.