Logo - springer
Slogan - springer

Life Sciences | Stem Cell Therapy in Lysosomal Storage Diseases

Stem Cell Therapy in Lysosomal Storage Diseases

Boelens, Jaap Jan, Wynn, Robert (Eds.)

2013, IX, 171 p. 6 illus., 3 illus. in color.

A product of Humana Press
Available Formats:

Springer eBooks may be purchased by end-customers only and are sold without copy protection (DRM free). Instead, all eBooks include personalized watermarks. This means you can read the Springer eBooks across numerous devices such as Laptops, eReaders, and tablets.

You can pay for Springer eBooks with Visa, Mastercard, American Express or Paypal.

After the purchase you can directly download the eBook file or read it online in our Springer eBook Reader. Furthermore your eBook will be stored in your MySpringer account. So you can always re-download your eBooks.


(net) price for USA

ISBN 978-1-4614-8357-1

digitally watermarked, no DRM

Included Format: PDF and EPUB

download immediately after purchase

learn more about Springer eBooks

add to marked items


Hardcover version

You can pay for Springer Books with Visa, Mastercard, American Express or Paypal.

Standard shipping is free of charge for individual customers.


(net) price for USA

ISBN 978-1-4614-8356-4

free shipping for individuals worldwide

usually dispatched within 3 to 5 business days

add to marked items

  • 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 field
  • Examines the current roles of treatments, including newer ones, and how they may be used in combination to treat these rare diseases
  • Provides state of the art treatment that can be implemented in daily practice and looks at possible long and short term future developments
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.

Content Level » Professional/practitioner

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

Related subjects » Cell Biology - Human Genetics - Life Sciences

Table of contents 

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.

Popular Content within this publication 



Read this Book on Springerlink

Services for this book

New Book Alert

Get alerted on new Springer publications in the subject area of Stem Cells.