Central Nervous System-directed AAV2/5-Mediated Gene Therapy Synergizes with Bone Marrow Transplantation in the Murine Model of Globoid-cell Leukodystrophy

This publication has 30 references indexed in Scilit:

• AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2
  Molecular Therapy, 2005
• AAV-Mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy
  Molecular Therapy, 2005
• Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements
  Gene Therapy, 2001
• Hematopoietic Stem-Cell Transplantation in Globoid-Cell Leukodystrophy
  New England Journal of Medicine, 1998
• Molecular genetics of Krabbe disease (globoid cell leukodystrophy): Diagnostic and clinical implications
  Human Mutation, 1997
• Murine model of genetic demyelinating disease: The twitcher mouse
  Microscopy Research and Technique, 1995
• Effect of bone marrow transplantation on enzyme levels and clinical course in the neurologically affected twitcher mouse.
  Journal of Clinical Investigation, 1988
• Prolonged Survival and Remyelination After Hematopoietic Cell Transplantation in the Twitcher Mouse
  Science, 1984
• The twitcher mouse: an enzymatically authentic model of human globoid cell leukodystrophy (Krabbe disease)
  Brain Research, 1980
• Globoid Cell Leucodystrophy (Krabbe's Disease): Deficiency of Galactocerebroside β-Galactosidase
  Proceedings of the National Academy of Sciences, 1970