Download or read book Phenotyping of Human iPSC derived Neurons written by Elizabeth D. Buttermore and published by Academic Press. This book was released on 2022-09-09 with total page 374 pages. Available in PDF, EPUB and Kindle. Book excerpt: Phenotyping of Human iPSC-derived Neurons: Patient-Driven Research examines the steps in a preclinical pipeline that utilizes iPSC-derived neuronal technology to better understand neurological disorders and identify novel therapeutics, also providing considerations and best practices. By presenting example projects that identify phenotypes and mechanisms relevant to autism spectrum disorder and epilepsy, this book allows readers to understand what considerations are important to assess at the start of project design. Sections address reproducibility issues and advances in technology at each stage of the pipeline and provide suggestions for improvement. From patient sample collection and proper controls to neuronal differentiation, phenotyping, screening, and considerations for moving to the clinic, these detailed descriptions of each stage of the pipeline will help everyone, regardless of stage in the pipeline. In recent years, drug discovery in the neurosciences has struggled to identify novel therapeutics for patients with varying indications, including epilepsy, chronic pain, and psychosis. Current treatment options for such patients are decades old and offer little relief with many side effects. One explanation for this lull in novel therapeutics is a lack of novel target identification for neurological disorders (and target identification requires exemplar preclinical data). To improve on the preclinical work that often relies on rodent modeling, the field has begun utilizing patient-derived induced pluripotent stem cells (iPSCs) to differentiate neurons in vitro for preclinical characterization of neurological disease and target identification. Discusses techniques and new technology for iPSC culturing and neuronal differentiation to establish best practices in the lab Outlines considerations for phenotypic assay development Provides information about the successes, failures, and implications of phenotyping and screening with iPSC-derived neurons Describes how human iPSC-derived neurons are being used for preclinical discovery research as well as the development of therapeutics utilizing hiPSC-derived neurons

Download or read book Leukemia Stem Cells written by César Cobaleda and published by Humana. This book was released on 2020-12-11 with total page 479 pages. Available in PDF, EPUB and Kindle. Book excerpt: The detailed volume aims to provide a comprehensive hands-on manual covering all the techniques involved in the cellular and molecular identification and characterization of both normal hematopoietic and leukemic stem cells, both from human patients and from mouse models of human leukemia. The book also covers the most frequently used experimental approaches for the generation of such stem cell-based models of human leukemia. 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 protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and comprehensive, Leukemia Stem Cells: Methods and Protocols serves as an ideal guide for researchers, both expert and novice, seeking to further our knowledge of this vital avenue of cancer research.

Download or read book Improving and Accelerating Therapeutic Development for Nervous System Disorders written by Institute of Medicine and published by National Academies Press. This book was released on 2014-02-06 with total page 107 pages. Available in PDF, EPUB and Kindle. Book excerpt: Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials.

Download or read book Comprehensive Medicinal Chemistry III written by and published by Elsevier. This book was released on 2017-06-03 with total page 4609 pages. Available in PDF, EPUB and Kindle. Book excerpt: Comprehensive Medicinal Chemistry III, Eight Volume Set provides a contemporary and forward-looking critical analysis and summary of recent developments, emerging trends, and recently identified new areas where medicinal chemistry is having an impact. The discipline of medicinal chemistry continues to evolve as it adapts to new opportunities and strives to solve new challenges. These include drug targeting, biomolecular therapeutics, development of chemical biology tools, data collection and analysis, in silico models as predictors for biological properties, identification and validation of new targets, approaches to quantify target engagement, new methods for synthesis of drug candidates such as green chemistry, development of novel scaffolds for drug discovery, and the role of regulatory agencies in drug discovery. Reviews the strategies, technologies, principles, and applications of modern medicinal chemistry Provides a global and current perspective of today's drug discovery process and discusses the major therapeutic classes and targets Includes a unique collection of case studies and personal assays reviewing the discovery and development of key drugs

Download or read book Genetics of Epilepsy written by and published by Elsevier. This book was released on 2014-09-04 with total page 346 pages. Available in PDF, EPUB and Kindle. Book excerpt: The book chapters cover different aspects of epilepsy genetics, starting with the "classical" concept of epilepsies as ion channel disorders. The second part of the book gives credit to the fact that by now non-ion channel genes are recognized as equally important causes of epilepsy. The concluding chapters are designed to offer the reader insight into current methods in epilepsy research. Each chapter is self-contained and deals with a selected topic of interest. Authors are the leading experts in the field of epilepsy research Book covers the most important aspects of epilepsy Interesting for both scientists and clinicians

Download or read book Targeting Ion Channels for Drug Discovery Emerging Challenges for High Throughput Screening Technologies written by Ciria Hernandez and published by Frontiers Media SA. This book was released on 2024-06-07 with total page 153 pages. Available in PDF, EPUB and Kindle. Book excerpt: Ligand and voltage-gated ion channels are highly regulated protein molecules that cross the cell membrane allowing ion flow from one side of the membrane to the other. They are ubiquitously expressed in human tissues and consist of one of the largest and best understood functional groups of proteins, with more than 400 members spanning nearly 1% of the human genome. They are involved in a variety of fundamental physiological processes, and their malfunction causes numerous diseases. In terms of the challenges faced in the effort to discover specific drugs in ancient and emerging diseases, ion channels are the third-largest class of target proteins after G-protein-coupled receptors (GPCRs) and kinases. 15% of small molecule drug targets have been reported to be voltage- or ligand-gated ion channels, resulting in approximately 150 new drug candidates in preclinical and clinical studies. Of the ion channel targeting drugs found on the market, these were identified more than a decade ago, and many of the current studies are at various stages of scientific approval. Overcoming these challenges has led the field of ion channel drug discovery to transform over the past 15 years through major advancements in genetic target detection, validation, structure-based drug design, and drug modeling of cell-based diseases.

Download or read book Celebrating the Diversity of Genetic Research to Dissect the Pathogenesis of Parkinson s Disease written by Soraya Bardien and published by Frontiers Media SA. This book was released on 2021-05-05 with total page 227 pages. Available in PDF, EPUB and Kindle. Book excerpt: We would like to acknowledge Prof Rejko Krueger, from University of Luxembourg, who acted as a coordinator contributing to the preparation of the proposal for this Research Topic as part of the GEoPD.

Download or read book Modeling Neuromuscular Diseases to Determine Molecular Drivers of Pathology and for Drug Discovery written by David Lee Mack and published by Frontiers Media SA. This book was released on 2022-11-14 with total page 195 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Neurodevelopmental Disorders written by Emanuel DiCicco-Bloom and published by Springer Nature. This book was released on 2020-06-24 with total page 307 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book contains a compendium of induced pluripotent stem cells (iPSCs) articles and reviews concerning state of the art technologies and how they are being applied to human neurodevelopmental disorders. With the establishment of effective technologies to produce iPSCs and their derivatives, like neural precursors, neurons, and glia, researchers have new platforms to study neurodevelopmental disorders. iPSC technology enables researchers to study how human neurons develop in individuals with neurodevelopmental disorders, providing an unparalleled opportunity to investigate their etiology. In turn, researchers have now begun to understand the underlying molecular and cellular pathways that contribute to human diseases. iPSCs technologies also provide an emerging tool for future translational studies and disease classification. The chapters will emphasize how among the diverse idiopathic and genetic disorders, there are common clinical as well as cellular and molecular phenotypes.

Download or read book Myoblast Transfer Therapy written by Robert C. Griggs and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 305 pages. Available in PDF, EPUB and Kindle. Book excerpt: I am pleased to introduce this volume on Myoblast Transfer Therapy on behalf of the Muscular Dystrophy Association and all of its Advisory Committees. The international conference which led to this volume brought together leading basic scientists and clinical investigators for the purpose of coordinating the development of this new field in the fight against muscular dystrophy. The Muscular Dystrophy Association is the nation's most rapidly growing voluntary health agency in terms of its programs of patient care, research, and professional and public education. Success is attributable to its National Chairman, Jerry Lewis, to its effective corporate membership, and to the many physicians and scientists who give their time freely to advise on policies, to review grant applications, and to participate in meetings such as this. I should like to acknowledge a large number of other individuals to whom we are indebted: the broad segment of the American public which continually and generously supports our spectrum of services. The Muscular Dystrophy Association, next year, should raise in excess of $115,000,000. These contributions are derived from more than 10 million American families. These families are not only pledging their money but expressing their hopes that we will find answers to the tragic problem of neuromuscular disease. We are confident that the fruits of this meeting will move the frontier of research forward toward that goal.

Download or read book Neurodevelopmental Disorders written by Bin Chen and published by Academic Press. This book was released on 2020-05-24 with total page 428 pages. Available in PDF, EPUB and Kindle. Book excerpt: Neurodevelopmental Disorders, the latest release in the Comprehensive Developmental Neuroscience series, presents the most thorough coverage available, addressing all aspects on how the nervous system and its components develop. This book brings together the latest research in this rapidly evolving field, with section editors discussing the technological advances that are enabling the pursuit of new research on brain development. This volume focuses on neurodevelopmental disorders in humans and experimental organisms. Particular attention is paid to the effects of abnormal development and on new psychiatric/neurological treatments being developed based on our increased understanding of developmental mechanisms. Features leading experts in various subfields as section editors and article authors Presents articles that have been peer reviewed to ensure accuracy, thoroughness and scholarship Covers disorders of the nervous system that arise through defects in neural development

Download or read book Basic Clinical Neuroscience written by Paul A. Young and published by Lippincott Williams & Wilkins. This book was released on 2008 with total page 422 pages. Available in PDF, EPUB and Kindle. Book excerpt: Basic Clinical Neuroscience offers medical and other health professions students a clinically oriented description of human neuroanatomy and neurophysiology. This text provides the anatomic and pathophysiologic basis for understanding neurologic abnormalities through concise descriptions of functional systems with an emphasis on medically important structures and clinically important pathways. It emphasizes the localization of specific anatomic structures and pathways with neurological deficits, using anatomy enhancing 3-D illustrations. Basic Clinical Neuroscience also includes boxed clinical information throughout the text, a key term glossary section, and review questions at the end of each chapter, making this book comprehensive enough to be an excellent Board Exam preparation resource in addition to a great professional training textbook. The fully searchable text will be available online at thePoint.

Download or read book Neural and Synaptic Defects in Autism Spectrum Disorders written by Hansen Wang and published by Frontiers Media SA. This book was released on 2015-07-07 with total page 287 pages. Available in PDF, EPUB and Kindle. Book excerpt: Autism spectrum disorders (ASDs) are a group of genetically and clinically heterogeneous neurodevelopmental disorders characterized by impaired reciprocal social interactions and communication, and restricted and repetitive patterns of behaviors and interests. Studies in genetics, neurobiology and systems biology are providing insights into the pathogenesis of ASDs. Investigation of neural and synaptic defects in ASDs not only sheds light on the molecular and cellular mechanisms that govern the function of the central nervous system, but may lead to the discovery of potential therapeutic targets for autism and other cognitive disorders. Our Research Topic which constitutes this e-book documents the recent development and ideas in the study of pathogenesis and treatment of ASDs, with an emphasis on syndromic disorders such as fragile X and Rett syndromes. In addition, model systems and methodological approaches with translational relevance to autism are covered herein. We hope that the Research Topic will enhance the global knowledge base in the autism research community and foster new research directions in autism related biology.

Download or read book Genome Editing in Drug Discovery written by Marcello Maresca and published by John Wiley & Sons. This book was released on 2022-03-29 with total page 355 pages. Available in PDF, EPUB and Kindle. Book excerpt: GENOME EDITING IN DRUG DISCOVERY A practical guide for researchers and professionals applying genome editing techniques to drug discovery In Genome Editing in Drug Discovery, a team of distinguished biologists delivers a comprehensive exploration of genome editing in the drug discovery process, with coverage of the technology’s history, current issues and techniques, and future perspectives and research directions. The book discusses techniques for disease modeling, target identification with CRISPR, safety studies, therapeutic editing, and intellectual property issues. The safety and efficacy of drugs and new target discovery, as well as next-generation therapeutics are also presented. Offering practical suggestions for practitioners and academicians involved in drug discovery, Genome Editing in Drug Discovery is a fulsome treatment of a technology that has become part of nearly every early step in the drug discovery pipeline. Selected contributions also include: A thorough introduction to the applications of CRISPRi and CRISPRa in drug discovery Comprehensive explorations of genome-editing applications in stem cell engineering and regenerative medicine Practical discussions of the safety aspects of genome editing with respect to immunogenicity and the specificity of CRISPR-Cas9 gene editing In-depth examinations of critical socio-economic and bioethical challenges in the CRISPR-Cas9 patent landscape Perfect for academic researchers and professionals in the biotech and pharmaceutical industries, Genome Editing in Drug Discovery will also earn a place in the libraries of medicinal chemists, biochemists, and molecular biologists.

Download or read book Chasing Men on Fire written by Stephen G. Waxman and published by MIT Press. This book was released on 2018-03-09 with total page 320 pages. Available in PDF, EPUB and Kindle. Book excerpt: A thirty-year quest, from genes to pain-signaling neurons to people with a rare genetic disorder that makes them feel they are on fire. Two soldiers, both with wounds injuring the same nerve, show very different responses: one is disabled by neuropathic pain, unable to touch the injured limb because even the lightest contact triggers excruciating discomfort; the other notices numbness but no pain at all. Could the difference lie in their genes? In this book, described in the foreword by Nobel Laureate James Rothman as “so well written that it reads like a detective novel,” Stephen Waxman recounts the search for a gene that controls pain—a search spanning more than thirty years and three continents. The story moves from genes to pain-signaling neurons that scream when they should be silent to people with a rare genetic disorder who feel they are on fire. Waxman explains that if pain-signaling neurons are injured by trauma or disease, they can become hyperactive and send pain signals to the brain even without external stimulus. Studying the hyperactive mutant pain gene in man on fire syndrome has pointed the way to molecules that produce pain more broadly within the general population, in the rest of us. Waxman's account of the many steps that led to discovery of the pain gene tells the story behind the science, of how science happens.

Download or read book Stem Cells in Aesthetic Procedures written by Melvin A. Shiffman and published by Springer. This book was released on 2014-07-21 with total page 815 pages. Available in PDF, EPUB and Kindle. Book excerpt: Interest in the use of stem cells in aesthetic procedures has been increasing rapidly, reflecting the widespread acknowledgment of the tremendous potential of stem cell fat transfer. This is, however, the first book to be devoted entirely to the subject. The book opens by reviewing the history of the development of pluripotent stem cells and the results of research into the biochemistry and physiology of stem cells. Adipose tissue anatomy and survival are discussed and the wide range of aesthetic procedures involving stem cell fat transfer are then described in detail. These procedures relate to the face, breast, buttocks, legs, hands, penis and Poland syndrome. In addition, potential risks and complications are identified. The book has been written by leading experts and will be an invaluable source of information for students, beginners and experienced surgeons in a range of specialties.

Download or read book New Oxford Textbook of Psychiatry written by John R. Geddes and published by Oxford University Press. This book was released on 2020-03-25 with total page 1472 pages. Available in PDF, EPUB and Kindle. Book excerpt: Over its two editions, The New Oxford Textbook of Psychiatry has come to be regarded as one of the most popular and trusted standard psychiatry texts among psychiatrists and trainees. Bringing together 146 chapters from the leading figures in the discipline, it presents a comprehensive account of clinical psychiatry, with reference to its scientific basis and to the patient's perspective throughout. The New Oxford Textbook of Psychiatry, Third Edition has been extensively re-structured and streamlined to keep pace with the significant developments that have taken place in the fields of clinical psychiatry and neuroscience since publication of the second edition in 2009. The new edition has been updated throughout to include the most recent versions of the two main classification systems—-the DSM-5 and the ICD-11—-used throughout the world for the diagnosis of mental disorders. In the years since publication of the first edition, many new and exciting discoveries have occurred in the biological sciences, which are having a major impact on how we study and practise psychiatry. In addition, psychiatry has fostered closer ties with philosophy, and these are leading to healthy discussions about how we should diagnose and treat mental illness. This new edition recognises these and other developments. Throughout, accounts of clinical practice are linked to the underlying science, and to the evidence for the efficacy of treatments. Physical and psychological treatments, including psychodynamic approaches, are covered in depth. The history of psychiatry, ethics, public health aspects, and public attitudes to psychiatry and to patients are all given due attention.