Stem Cell Therapy in Lysosomal Storage Diseases

• Συγγραφή απο Οργανισμό/Αρχή: SpringerLink (Online service)
• Άλλοι συγγραφείς: Boelens, Jaap Jan (Επιμελητής έκδοσης), Wynn, Robert (Επιμελητής έκδοσης)
• Μορφή: Ηλεκτρονική πηγή Ηλ. βιβλίο
• Γλώσσα: English
• Έκδοση: New York, NY : Springer New York : Imprint: Humana Press, 2013.
• Σειρά: Stem Cell Biology and Regenerative Medicine,
• Θέματα: Life sciences.; Human genetics.; Cell biology.; Stem cells.; Life Sciences.; Stem Cells.; Cell Biology.; Human Genetics.

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...

Πίνακας περιεχομένων:

• 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.