Phenotyping Of Human Ipsc Derived Neurons
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Phenotyping Of Human Ipsc Derived Neurons
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Author : Elizabeth D. Buttermore
language : en
Publisher: Academic Press
Release Date : 2022-09-09
Phenotyping Of Human Ipsc Derived Neurons written by Elizabeth D. Buttermore and has been published by Academic Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-09-09 with Medical categories.
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
Comprehensive Medicinal Chemistry Iii
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Author :
language : en
Publisher: Elsevier
Release Date : 2017-06-03
Comprehensive Medicinal Chemistry Iii written by and has been published by Elsevier this book supported file pdf, txt, epub, kindle and other format this book has been release on 2017-06-03 with Technology & Engineering categories.
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
Cell Therapy
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Author : Nguyen Thanh Liem
language : en
Publisher: Springer Nature
Release Date : 2025-05-12
Cell Therapy written by Nguyen Thanh Liem and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2025-05-12 with Science categories.
This book provides an overview of cell-based therapy for human diseases including the definition, history, and clinical applications of human stem cells and their use in regenerative medicine. It covers human pluripotent stem cells (human embryonic stem cells and human induced pluripotent stem cells), mesenchymal stem/stromal cells, and hematopoietic stem cells, bone marrow mononuclear cells. In terms of clinical applications, this book also provides an update on recent human trials using these cells to treat various diseases, including neurological disorders, pulmonary dysfunctions, metabolic/endocrine-related diseases, frailty, and cancer treatment. In addition, it discusses the authors’ clinical trial experiences related to these conditions in a clinical setting to provide additional insight into regenerative medicine, especially cell-based therapy. The field of regenerative medicine has witnessed tremendous advancement in stem cell research and therapy in recent years, with thousands of preclinical studies supporting thousands of human clinical trials conducted worldwide. Emerging evidence in regenerative medicine and stem cell research suggests the treatments are safe, whereas their therapeutic potential, effectiveness, and mode of action require deeper investigation. It is important to introduce the science behind stem cell-based therapy to patients, clinicians, and researchers to further enhance understanding of responsible clinical conduct and so to educate and thus prevent the further emergence of unregulated, deceitful stem cell clinics solely operating for the sake of profit.
Targeting Ion Channels For Drug Discovery Emerging Challenges For High Throughput Screening Technologies
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Author : Ciria Hernandez
language : en
Publisher: Frontiers Media SA
Release Date : 2024-06-07
Targeting Ion Channels For Drug Discovery Emerging Challenges For High Throughput Screening Technologies written by Ciria Hernandez and has been published by Frontiers Media SA this book supported file pdf, txt, epub, kindle and other format this book has been release on 2024-06-07 with Science categories.
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.
Celebrating The Diversity Of Genetic Research To Dissect The Pathogenesis Of Parkinson S Disease
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Author : Soraya Bardien
language : en
Publisher: Frontiers Media SA
Release Date : 2021-05-05
Celebrating The Diversity Of Genetic Research To Dissect The Pathogenesis Of Parkinson S Disease written by Soraya Bardien and has been published by Frontiers Media SA this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-05-05 with Medical categories.
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.
Modeling Neuromuscular Diseases To Determine Molecular Drivers Of Pathology And For Drug Discovery
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Author : David Lee Mack
language : en
Publisher: Frontiers Media SA
Release Date : 2022-11-14
Modeling Neuromuscular Diseases To Determine Molecular Drivers Of Pathology And For Drug Discovery written by David Lee Mack and has been published by Frontiers Media SA this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-11-14 with Science categories.
Neurodevelopmental Disorders
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Author : Bin Chen
language : en
Publisher: Academic Press
Release Date : 2020-05-24
Neurodevelopmental Disorders written by Bin Chen and has been published by Academic Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-05-24 with Medical categories.
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
Neurodevelopmental Disorders
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Author : Emanuel DiCicco-Bloom
language : en
Publisher: Springer Nature
Release Date : 2020-06-24
Neurodevelopmental Disorders written by Emanuel DiCicco-Bloom and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-06-24 with Medical categories.
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.
Genome Editing In Drug Discovery
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Author : Marcello Maresca
language : en
Publisher: John Wiley & Sons
Release Date : 2022-03-29
Genome Editing In Drug Discovery written by Marcello Maresca and has been published by John Wiley & Sons this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-03-29 with Science categories.
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.
Chasing Men On Fire
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Author : Stephen G. Waxman
language : en
Publisher: MIT Press
Release Date : 2018-03-09
Chasing Men On Fire written by Stephen G. Waxman and has been published by MIT Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-03-09 with Medical categories.
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.