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Science and medicine have provided us with clues to the treatment of a few genetic diseases, although by their very nature they have never been considered curable. But, as William R. Clark shows, that is about to change through one of the most profound revolutions in modern medicine: gene therapy, a branch of the new field of molecular medicine. Clark takes us to the laboratories which have been able to isolate human genes, to make billions of copies of them, and to reintroduce healthy genes into unfortunate individuals who have inherited damaged or functionless genes.

Gene therapy --- Therapy, Gene --- Genetic engineering --- Therapeutics

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Gene therapy has emerged as a discipline in medicine that can provide treatments for diseases that have no other therapies available, save lives of patients for whom there is no other hope and replace suboptimal treatments with lasting cures. 'Fast Facts: Gene Therapy' provides an overview of the field, looking at the main vector systems used to transfer the therapeutic gene constructs, the molecular mechanisms and the history of gene therapy, as well as the safety and ethical considerations of this important advance. Multiple examples of diseases that are already successfully treated with gene therapy are given, with discussion of treatments that hold promise for the future. This book will be informative and of value to health professionals, researchers, students and anyone with an interest in this exciting and fast-moving area. Contents: • Principles of gene therapy • Gene therapy techniques • Ethical and safety considerations • Gene therapies with proven clinical efficacy • Genome editing • Research directions – the next wave of treatments

Gene therapy. --- Therapy, Gene --- Genetic engineering --- Therapeutics

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The aim of our book is to provide a detailed discussion of gene therapy application in human diseases. The book brings together major approaches: (1) Gene therapy in blood and vascular system, (2) Gene therapy in orthopedics, (3) Gene therapy in genitourinary system, (4) Gene therapy in other diseases. This source will make clinicians and researchers comfortable with the potential and problems of gene therapy application.

Gene therapy. --- Therapy, Gene --- Genetic engineering --- Therapeutics --- Medical genetics

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This third edition provides in-depth knowledge on the delivery of naked DNA and small-interfering RNA (siRNA) to the targeted microorganism, mammalian single cells, tissues, and animals for prevention and treatment of disease. It builds on the success of the first edition and on the progress made in siRNA delivery and DNA vaccines for large animals as well as discovery of electroporation applications for the fragile tissues and for internal organs. 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 easily accessible, Electroporation Protocols: Microorganism, Mammalian System, and Nanodevice, Third Edition aims to be an invaluable resource for investigators both in and outside of this field. .

Gene therapy. --- Gene Therapy. --- Therapy, Gene --- Genetic engineering --- Therapeutics

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Human genetics --- Gene therapy --- Gentherapie --- Therapie [Gen] --- Therapy [Gene ] --- Thérapie génique