Description |
1 online resource (ix, 171 pages) : illustrations (some color) |
Series |
Stem Cell Biology and Regenerative Medicine, 2196-8985 |
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Stem cell biology and regenerative medicine, 2196-8985
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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
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Stem cells -- Therapeutic use
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Stem cells -- Transplantation.
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Genetic Therapy -- methods
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Stem Cell Transplantation
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Stem Cells -- metabolism
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Enzyme Replacement Therapy -- methods
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Lysosomal Storage Diseases -- therapy
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Lysosomal Storage Diseases -- physiopathology
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HEALTH & FITNESS -- Diseases -- General.
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MEDICAL -- Clinical Medicine.
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MEDICAL -- Diseases.
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MEDICAL -- Evidence-Based Medicine.
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MEDICAL -- Internal Medicine.
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Stem cells -- Transplantation
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Lysosomal storage diseases -- Treatment
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Form |
Electronic book
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Author |
Boelens, Jaap Jan, editor
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Wynn, Robert F. (Robert Francis), editor.
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ISBN |
9781461483571 |
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1461483573 |
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