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Hematopoietic stem cell gene therapy for the treatment of metachromatic leukodystrophy

Bibliographic Details
Main Author: Biffi, Alessandra
Format: eBook
Language:English
Published: London Henry Stewart Talks 2014, 2014
Series:Gene transfer and gene therapy
Subjects:
Genetic Therapy
Lentivirus
Brain / Diseases / Genetic Aspects
Microglia
Gene Therapy
Hematopoietic Stem Cell Transplantation
Medical Genetics
Genetics, Medical
Leukodystrophy, Metachromatic / Therapy
Leukodystrophy, Metachromatic / Genetics
Hematopoietic Stem Cells / Metabolism
Virus Integration / Genetics
Stem Cells / Therapeutic Use
Online Access:
Collection: Henry Stewart Talks - Collection details see MPG.ReNa
Table of Contents:
  • Metachromatic Leukodystrophy (MLD)
  • Hematopoietic stem cell therapy (HCT) for lysosomal storage disorders (LSDs)
  • Improving HCT efficacy
  • The role of brain pre-conditioning
  • Mechanisms of microglia turnover
  • Gene therapy (GT) & enzyme release
  • Gene therapy in murine models
  • HSC gene therapy for MLD
  • Lentivirus integration site analysis
  • Stem cells analysis
  • ARSA activity and delivery to the CNS
  • Cerebral and peripheral demyelination
  • Motor function testing (GMFM)

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