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E-book

Title Stem cell therapy in Lysosomal storage diseases / Jaap Jan Boelens, Robert Wynn, editors
Published New York, NY : Humana Press, 2013

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Description 1 online resource (ix, 171 pages) : illustrations (some color)
Series Stem Cell Biology and Regenerative Medicine, 2196-8985
Stem cell biology and regenerative medicine, 2196-8985
Contents 1. Lysosomal Diseases and Therapeutic Options: An Overview -- 2. Alternative Treatment Options: Enzyme Replacement and Small Molecule Therapies -- 3. Hematopoietic Cell Transplantation in Inborn Errors of Metabolism -- 4. Allogeneic Hematopoietic Stem Cell Transplantation in Lysosomal Storage Disorders: Long Term Neurodevelopmental and Somatic Outcome -- 5. Hematopoietic Stem Cell Gene Therapy for Lysosomal Storage Disorders: Expected Benefits and Limitations -- 6. Alternative Future Therapies for Lysosomal Storage Diseases: Embryonic Stem Cell- and Induced Pluripotent Stem Cell Therapy -- 7. New Directions and Conclusions
Summary 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
Notes Online resource; title from PDF title page (SpringerLink, viewed October 14, 2013)
Subject Lysosomal storage diseases -- Treatment
Stem cells -- Therapeutic use
Stem cells -- Transplantation.
Genetic Therapy -- methods
Stem Cell Transplantation
Stem Cells -- metabolism
Enzyme Replacement Therapy -- methods
Lysosomal Storage Diseases -- therapy
Lysosomal Storage Diseases -- physiopathology
HEALTH & FITNESS -- Diseases -- General.
MEDICAL -- Clinical Medicine.
MEDICAL -- Diseases.
MEDICAL -- Evidence-Based Medicine.
MEDICAL -- Internal Medicine.
Stem cells -- Transplantation
Lysosomal storage diseases -- Treatment
Form Electronic book
Author Boelens, Jaap Jan, editor
Wynn, Robert F. (Robert Francis), editor.
ISBN 9781461483571
1461483573