Stem Cell Therapy in Lysosomal Storage Diseases

  • Jaap Jan Boelens
  • Robert Wynn

Part of the Stem Cell Biology and Regenerative Medicine book series (STEMCELL)

Table of contents

  1. Front Matter
    Pages i-ix
  2. Eveline J. Langereis, Frits A. Wijburg
    Pages 1-20
  3. Jaap J. Boelens, Paul Szabolcs, Rob Wynn
    Pages 37-61
  4. Maria L. Escolar, Mieke Aldenhoven
    Pages 63-125
  5. Robert Wynn, Jaap Jan Boelens
    Pages 159-166
  6. Back Matter
    Pages 167-171

About this book


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.


Enzyme replacement therapy Gene therapy Hematopoietic cell transplantation Inborn errors of metabolism Peroxisomes

Editors and affiliations

  • Jaap Jan Boelens
    • 1
  • Robert Wynn
    • 2
  1. 1.University Medical Center Utrecht Wilhelmina Childrens HospitalUtrechtThe Netherlands
  2. 2.Royal Manchester Children's HospitalManchesterUnited Kingdom

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