Phenotyping Of Human Ipsc Derived Neurons


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Phenotyping of Human iPSC-derived Neurons


Phenotyping of Human iPSC-derived Neurons

Author: Elizabeth D. Buttermore

language: en

Publisher: Academic Press

Release Date: 2022-09-09


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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


Comprehensive Medicinal Chemistry III

Author:

language: en

Publisher: Elsevier

Release Date: 2017-06-03


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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


Cell Therapy

Author: Nguyen Thanh Liem

language: en

Publisher: Springer Nature

Release Date: 2025-05-12


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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.