TY - BOOK AU - Boelens,Jaap Jan AU - Wynn,Robert ED - SpringerLink (Online service) TI - Stem Cell Therapy in Lysosomal Storage Diseases T2 - Stem Cell Biology and Regenerative Medicine, SN - 9781461483571 AV - QH588.S83 U1 - 571.6 23 PY - 2013/// CY - New York, NY PB - Springer New York, Imprint: Humana Press KW - Life sciences KW - Human genetics KW - Cell biology KW - Stem cells KW - Life Sciences KW - Stem Cells KW - Cell Biology KW - Human Genetics KW - Electronic books KW - local N1 - 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 N2 - 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 UR - http://ezproxy.alfaisal.edu/login?url=http://dx.doi.org/10.1007/978-1-4614-8357-1 ER -