Concluding Comments and Future Directions

  • Robert WynnEmail author
  • Jaap Jan Boelens
Part of the Stem Cell Biology and Regenerative Medicine book series (STEMCELL)


The chapters of this book describe enormous, almost unbelievable, progress in the last few years in the field of the cellular therapy of lysosomal storage disorders (LSDs). We have learnt much about the diseases themselves whilst we have made parallel great progress in the clinical management of affected individuals. In these concluding notes we, the editors, wish to both summarise the key advances that have been made in this field of cellular therapy of LSD and speculate upon the future of the field in the coming years and decades. Furthermore we wish to indicate how all us in scientific and clinical fields—editors, contributing authors and readers of this book—must act together in order to effect this future.


Enzyme Replacement Therapy Haematopoietic Stem Cell Transplant Cellular Therapy Lysosomal Storage Disorder Substrate Accumulation 
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Copyright information

© Springer Science+Business Media New York 2013

Authors and Affiliations

  1. 1.Royal Manchester Children’s HospitalManchesterUK
  2. 2.University Medical Center Utrecht, Wilhelmina Children’s HospitalUtrechtThe Netherlands

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