Stem Cell Therapy in Lysosomal Storage Diseases

1. Lysosomal Diseases and Therapeutic Options: An Overview
Eveline J. Langereis, Frits A. Wijburg

2. Alternative Treatment Options: Enzyme Replacement and Small Molecule Therapies
Simon Jones

3. Hematopoietic Cell Transplantation in Inborn Errors of Metabolism
Jaap J. Boelens, Paul Szabolcs, Rob Wynn

4. Leukodystrophies and Lysosomal Storage Disorders
Maria L. Escolar, Mieke Aldenhoven

5. Hematopoietic Stem Cell Gene Therapy for Lysosomal Storage Disorders: Expected Benefits and Limitations
Alessandra Biffi, Ilaria Visigalli

6. Alternative Future Therapies for Lysosomal Storage Diseases: Embryonic Stem Cell- and Induced Pluripotent Stem Cell Therapy
Arie Koen Braat, Paul J. Coffer, Niels Geijsen

7. Concluding Comments and Future Directions
Robert Wynn, Jaap Jan Boelens

Avainsanat: Life Sciences, Stem Cells, Cell Biology, Human Genetics