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Lysosomal Diseases and Therapeutic Options: An Overview

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Book cover Stem Cell Therapy in Lysosomal Storage Diseases

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

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Abstract

Lysosomal diseases cover a wide spectrum of inborn metabolic disorders and are characterized by a remarkable variability in pathophysiology and clinical presentation. This first chapter presents an overview of the natural history and biochemical characteristics of lysosomal storage disorders and some of the developments in therapy that have been made in the past decades and a brief look at potential future therapies.

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Author information

Authors and Affiliations

  1. Department of Pediatrics, Academic Medical Center, Amsterdam, The Netherlands

    Eveline J. Langereis & Frits A. Wijburg

Authors
  1. Eveline J. Langereis
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  2. Frits A. Wijburg
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Correspondence to Frits A. Wijburg .

Editor information

Editors and Affiliations

  1. University Medical Center Utrecht Wilhelmina Childrens Hospital, Utrecht, The Netherlands

    Jaap Jan Boelens

  2. Royal Manchester Children's Hospital, Manchester, United Kingdom

    Robert Wynn

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Langereis, E.J., Wijburg, F.A. (2013). Lysosomal Diseases and Therapeutic Options: An Overview. In: Boelens, J., Wynn, R. (eds) Stem Cell Therapy in Lysosomal Storage Diseases. Stem Cell Biology and Regenerative Medicine. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4614-8357-1_1

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