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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.

Cell organelles. --- Lysosomal storage diseases. --- Organelles. --- Peroxisomal disorders. --- Lysosomal storage diseases --- Stem cells --- Cell Transplantation --- Metabolic Phenomena --- Cells --- Enzyme Therapy --- Metabolism, Inborn Errors --- Investigative Techniques --- Anatomy --- Analytical, Diagnostic and Therapeutic Techniques and Equipment --- Phenomena and Processes --- Transplantation --- Drug Therapy --- Genetic Diseases, Inborn --- Congenital, Hereditary, and Neonatal Diseases and Abnormalities --- Therapeutics --- Surgical Procedures, Operative --- Diseases --- Methods --- Enzyme Replacement Therapy --- Stem Cell Transplantation --- Stem Cells --- Metabolism --- Lysosomal Storage Diseases --- Biology --- Health & Biological Sciences --- Cytology --- Treatment --- Therapeutic use --- Stem cells. --- Colony-forming units (Cells) --- Mother cells --- Progenitor cells --- Cell storage disorders --- Lysosomal disorders --- Lysosomal enzyme disorders --- Storage diseases, Lysosomal --- Life sciences. --- Human genetics. --- Cell biology. --- Life Sciences. --- Stem Cells. --- Cell Biology. --- Human Genetics. --- Metabolism, Inborn errors of --- Cytology. --- Genetics --- Heredity, Human --- Human biology --- Physical anthropology --- Cell biology --- Cellular biology --- Cytologists