Overview
- 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
Part of the book series: Stem Cell Biology and Regenerative Medicine (STEMCELL)
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Table of contents (7 chapters)
Keywords
About this book
Editors and Affiliations
About the editors
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 conditionsthan in any other European medical center.
Bibliographic Information
Book Title: Stem Cell Therapy in Lysosomal Storage Diseases
Editors: Jaap Jan Boelens, Robert Wynn
Series Title: Stem Cell Biology and Regenerative Medicine
DOI: https://doi.org/10.1007/978-1-4614-8357-1
Publisher: Humana New York, NY
eBook Packages: Biomedical and Life Sciences, Biomedical and Life Sciences (R0)
Copyright Information: Springer Science+Business Media New York 2013
Hardcover ISBN: 978-1-4614-8356-4Published: 11 October 2013
Softcover ISBN: 978-1-4939-4888-8Published: 23 August 2016
eBook ISBN: 978-1-4614-8357-1Published: 10 October 2013
Series ISSN: 2196-8985
Series E-ISSN: 2196-8993
Edition Number: 1
Number of Pages: IX, 171
Number of Illustrations: 3 b/w illustrations, 3 illustrations in colour
Topics: Stem Cells, Cell Biology, Human Genetics