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 t...

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Bibliographic Details
Call Number:Libro Electrónico
Corporate Author: SpringerLink (Online service)
Other Authors: Boelens, Jaap Jan (Editor), Wynn, Robert (Editor)
Format: Electronic eBook
Language:Inglés
Published: New York, NY : Springer New York : Imprint: Humana, 2013.
Edition:1st ed. 2013.
Series:Stem Cell Biology and Regenerative Medicine,
Subjects:
Stem cells.
Cytology.
Medical genetics.
Stem Cell Biology.
Cell Biology.
Medical Genetics.
Online Access:Texto Completo
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.

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