Stem Cell Therapy in Lysosomal Storage Diseases

Book Overview

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.

• Format: Paperback
• Language: English
• ISBN: 1493948881
• ISBN13: 9781493948888
• Release Date: August 2016
• Publisher: Humana
• Length: 171 Pages
• Weight: 0.59 lbs.
• Dimensions: 0.4" x 6.1" x 9.2"