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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
- Cover
- Title Page
- Copyright
- Contents
- List of abbreviations
- Glossary
- Introduction
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Principles of gene therapy
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History of genetic therapy
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Steps for development of gene therapy
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Analysis of the gene, gene product and related biological pathways
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Strategies for correcting the pathogenic gene
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Route of product delivery and tissue accessibility
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Preclinical translational studies
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Disease candidates for gene therapy
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Gene therapy techniques
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Loss-of-function mutations
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Gain-of-function mutations
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Complex diseases
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Somatic versus germline gene therapy
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Ex vivo application
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In vivo application
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Vectors
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Delivery using non-viral vectors
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Delivery using viral vectors
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Choice of viral vector
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Improvements needed
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Ethical and safety considerations
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Virus-related concerns
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Potential for immune responses
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Lack of specificity in gene transfer or cell targeting
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Germline gene therapy – a controversial topic
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Cost of gene therapies
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Gene therapies with proven clinical efficacy
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Hereditary blindness
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Neuromuscular disorder: spinal muscular atrophy
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Lysosomal storage disorder: metachromatic leukodystrophy
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Primary immune deficiencies: severe combined immune deficiencies
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Hematologic disorder: hemophilia
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Cancer gene therapy
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Genome editing
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Key principles and techniques
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Target diseases
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Research directions – the next wave of treatments
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Neuromuscular and neurological disorders
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Lysosomal storage disorders
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Muscle disorders
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Hemoglobinopathies
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- Useful resources
- Index
- 出版地 : 德國
- 語言 : 德文
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