Stem Cell Therapy in Lysosomal Storage Diseases
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.
Offers multidisciplinary input – from the clinic and the laboratory – into the management of LSD using cellular therapies, summarizing the recent and enormous changes in this fieldExamines the current roles of treatments, including newer ones, and how they may be used in combination to treat these rare diseasesProvides state of the art treatment that can be implemented in daily practice and looks at possible long and short term future developments