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Cancer --- Gene therapy

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The clinical applications of human gene therapy have been particularly fruitful in oncology, and in the last few decades there has been explosive growth in understanding of the genetic lesions leading to neoplasia. This volume in the series Cancer: Clinical Science in Practice reviews progress in the basic and clinical science of gene therapy in oncology, and looks forward to future developments. It considers what has worked and what has not in the fast-evolving field of gene therapy, drawing on laboratory studies and clinical trials, including the work of the contributors themselves. Topical and authoritative, volumes in this series are intended for a wide audience of clinicians and researchers with an interest in the applications of biomedical science to the understanding and management of cancer.

Cancer --- Gene therapy.

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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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Gene therapy. --- Musculoskeletal system --- Orthopedics. --- Sports medicine. --- Diseases --- Gene therapy.

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"On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop"--Publisher's description

Nervous system --- Diseases --- Gene therapy.