Stem Cell Therapy in Lysosomal Storage Diseases

✍ Scribed by Eveline J. Langereis, Frits A. Wijburg (auth.), Jaap Jan Boelens, Robert Wynn (eds.)

Publisher: Humana Press
Year: 2013
Tongue: English
Leaves: 178
Series: Stem Cell Biology and Regenerative Medicine
Edition: 1
Category: Library

No coin nor oath required. For personal study only.

✦ Synopsis

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.

✦ Table of Contents

Front Matter....Pages i-ix
Lysosomal Diseases and Therapeutic Options: An Overview....Pages 1-20
Alternative Treatment Options: Enzyme Replacement and Small Molecule Therapies....Pages 21-35
Hematopoietic Cell Transplantation in Inborn Errors of Metabolism....Pages 37-61
Leukodystrophies and Lysosomal Storage Disorders....Pages 63-125
Hematopoietic Stem Cell Gene Therapy for Lysosomal Storage Disorders: Expected Benefits and Limitations....Pages 127-138
Alternative Future Therapies for Lysosomal Storage Diseases: Embryonic Stem Cell- and Induced Pluripotent Stem Cell Therapy....Pages 139-158
Concluding Comments and Future Directions....Pages 159-166
Back Matter....Pages 167-171

✦ Subjects

Stem Cells; Cell Biology; Human Genetics

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