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Titlebook: Rare Diseases; Integrative PPPM App Meral Özgüç Book 2015 Springer Science+Business Media Dordrecht 2015 Invivo disease models.Personalized

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发表于 2025-3-21 19:49:28 | 显示全部楼层 |阅读模式
书目名称Rare Diseases
副标题Integrative PPPM App
编辑Meral Özgüç
视频video
概述State of the art of emerging genomics technologies for diagnosis.New insights into personalized therapy modalities.Serves as curriculum material for paradigm change towards predictive, preventive and
丛书名称Advances in Predictive, Preventive and Personalised Medicine
图书封面Titlebook: Rare Diseases; Integrative PPPM App Meral Özgüç Book 2015 Springer Science+Business Media Dordrecht 2015 Invivo disease models.Personalized
描述This book focuses on predictive, preventative and personalized medicine (PPPM) and how it is related to the healthcare of rare diseases. Readers will discover how advanced rare diseases healthcare provides an excellent “proof-of-principles” for the personalisation of healthcare systems on a global scale. Chapters look at national plans for rare disease, at biobanking, gene identification, rare cancers, virus gene therapy , induced pluripotency for cell therapy amongst other topics. There is a chapter dedicated to personalized medicine for hereditary deafness and another exploring the complexity of genotype-phenotype correlations. Specific diseases such as Fabry‘s, Gauchers and mitochondrial cytopathies are highlighted and we look at enzyme replacement therapy in lysosomal storage diseases. This work is part of a series, produced with the involvement of the European Association for Predictive, Preventive and Personalised Medicine. The series focusses on the concept of an integrative medical approach by PPPM. This volume is dedicated to all aspects related to the prediction, prevention and personalised treatments of rare diseases, and in doing so it explores developments relevant to
出版日期Book 2015
关键词Invivo disease models; Personalized medicine; Personalized therapies; Personel genome based diagnosis; R
版次1
doihttps://doi.org/10.1007/978-94-017-9214-1
isbn_softcover978-94-024-0802-7
isbn_ebook978-94-017-9214-1Series ISSN 2211-3495 Series E-ISSN 2211-3509
issn_series 2211-3495
copyrightSpringer Science+Business Media Dordrecht 2015
The information of publication is updating

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Adeno-Associated Virus Gene Therapy and Its Application to the Prevention and Personalised Treatmenis applied to rare diseases, the first section of this chapter aims to illustrate how gene therapy aligns to the principles of PPPM whereas the second part of the chapter intends to provide an in depth review of the developments in the AAV field that underpin the use of these viruses as gene therapy delivery systems.
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National Plans on Rare Diseases,specific features and approaches. It critically assesses the national achievements in this field, drawing conclusions to further strengthen the process of planning, implementing and evaluating RD national policies..A national plan for RD can be defined as an official strategic public health document
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,Biobanking for Rare Diseases – Impact on Personalised Medicine,ee the rights of stakeholders. Due to their medical and scientific expertise, they offer collections of human biological resources that meet with research project requirements. Because of their secure storage capacities, biobanks are front-line actors in collection management by enabling their devel
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Personalized Medicine for Hereditary Deafness,s. While the clinical phenotype, deafness, is shared and similar across different individuals, the substantial genetic heterogeneity makes the genetic etiology in individual cases rare. Identification of genetic causes has been central to a growing body of knowledge related to diagnostic tools, gene
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