Stem Cell Therapy in Lysosomal Storage Diseases [Paperback]

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.This volume discusses the history, current practice and future perspectives of stem cell in inborn errors of metabolism and provides an international perspective on progress, limitations, and future directions in the field.

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.

Jaap J. Boelens (1970) is a paediatrician  oncologist. Medicine (Utrecht 1996), PhD in Amsterdam (UVA; 1999), trained as a paediatrician in Leiden/Den Haag (2004) followed by completion of clinical fellowship in immunology (2006; Utrecht) and oncology (2011; Rotterdam). His clinical sub-specialization is Hematopoietic Cell Transplantation (HCT) and he works as a consultant in HCT at the UMC Utrecht since 2006. He has a special interest in rare-diseases (as indication for HCT) in particular lysosomal storage diseases and finding strategieslS²