Stem Cell Therapy in Lysosomal Storage Diseases
Editors: Boelens, Jaap Jan, Wynn, Robert (Eds.)
Free Preview- Offers multidisciplinary input – from the clinic and the laboratory – into the management of LSD using cellular therapies, summarizing the recent and enormous changes in this field
- Examines the current roles of treatments, including newer ones, and how they may be used in combination to treat these rare diseases
- Provides state of the art treatment that can be implemented in daily practice and looks at possible long and short term future developments
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- About this book
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Stem Cell therapy for lysosomal diseases (LSDs) is developing rapidly. This volume discusses the history, current practice and future perspectives of stem cells in inborn errors of metabolism (IEM) and provides an international perspective on progress, limitations, and future directions (e.g. gene therapy, iPS, ES) in the field. Beginning with an overview of these diseases, the book covers the breadth of this topic from treatment options, bone marrow transplantation, and alternative treatment options, through long-term outcomes and future perspectives.
- About the authors
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Jaap J. Boelens (1970) is a paediatrician – oncologist. Medicine (Utrecht 1996), PhD in Amsterdam (UVA; 1999), trained as a paediatrician in Leiden/Den Haag (2004) followed by completion of clinical fellowship in immunology (2006; Utrecht) and oncology (2011; Rotterdam). His clinical sub-specialization is Hematopoietic Cell Transplantation (HCT) and he works as a consultant in HCT at the UMC Utrecht since 2006. He has a special interest in rare-diseases (as indication for HCT) in particular “lysosomal storage diseases” and finding strategies to get better disease control (in malignant diseases). In close collaboration with dr Rob Wynn, the EBMT, EUROCORD and the CIBMTR he was PI of various international studies on the outcomes of HCT in “lysosomal storage diseases.” These studies has let to better international collaboration, guidelines and improved survival. He aims to further improve the outcomes of cellular-therapy for this group of patients, within the international network, especially focusing on the “residual disease burden,” which is present in almost all patients.
Dr. Robert Wynn is currently Consultant Pediatric Hematologist and Director of the Blood and Marrow Transplant Unit at the Royal Manchester Children's Hospital. He qualified in Medicine from Cambridge University and undertook further Medical, Pediatric and Hematology training in Cardiff, Manchester and Toronto. He is a both a member of the Royal College of Physicians and a fellow of the Royal College of Pathologists. He looks after children with malignant and non malignant blood disorders as a laboratory and a clinical hematologist.
Dr. Wynn's clinical and research focus is in metabolic disease transplant and was stimulated by Ed Wraith and his team at the Willink Unit at Royal Manchester Children's Hospital with its huge interest in lysosomal storage diseases and in which more transplants have been performed for these conditions than in any other European medical center.
- Table of contents (7 chapters)
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Lysosomal Diseases and Therapeutic Options: An Overview
Pages 1-20
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Alternative Treatment Options: Enzyme Replacement and Small Molecule Therapies
Pages 21-35
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Hematopoietic Cell Transplantation in Inborn Errors of Metabolism
Pages 37-61
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Leukodystrophies and Lysosomal Storage Disorders
Pages 63-125
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Hematopoietic Stem Cell Gene Therapy for Lysosomal Storage Disorders: Expected Benefits and Limitations
Pages 127-138
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Table of contents (7 chapters)
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Bibliographic Information
- Bibliographic Information
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- Book Title
- Stem Cell Therapy in Lysosomal Storage Diseases
- Editors
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- Jaap Jan Boelens
- Robert Wynn
- Series Title
- Stem Cell Biology and Regenerative Medicine
- Copyright
- 2013
- Publisher
- Humana Press
- Copyright Holder
- Springer Science+Business Media New York
- eBook ISBN
- 978-1-4614-8357-1
- DOI
- 10.1007/978-1-4614-8357-1
- Hardcover ISBN
- 978-1-4614-8356-4
- Softcover ISBN
- 978-1-4939-4888-8
- Series ISSN
- 2196-8985
- Edition Number
- 1
- Number of Pages
- IX, 171
- Number of Illustrations
- 3 b/w illustrations, 3 illustrations in colour
- Topics
