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Mechanisms of demyelination and neurodegeneration in globoid cell leukodystrophy
Globoid cell leukodystrophy (GLD), also known as Krabbe disease, is a lysosomal storage
disorder causing extensive demyelination in the central and peripheral nervous systems …
disorder causing extensive demyelination in the central and peripheral nervous systems …
[HTML][HTML] Nanomedicines to treat rare neurological disorders: The case of Krabbe disease
The brain remains one of the most challenging therapeutic targets due to the low and
selective permeability of the blood–brain barrier and complex architecture of the brain …
selective permeability of the blood–brain barrier and complex architecture of the brain …
Isolation of amniotic stem cell lines with potential for therapy
Stem cells capable of differentiating to multiple lineages may be valuable for therapy. We
report the isolation of human and rodent amniotic fluid–derived stem (AFS) cells that express …
report the isolation of human and rodent amniotic fluid–derived stem (AFS) cells that express …
Intracarotid injection of fluorescence activated cell-sorted CD49d-positive neural stem cells improves targeted cell delivery and behavior after stroke in a mouse stroke …
R Guzman, A De Los Angeles, S Cheshier, R Choi… - Stroke, 2008 - ahajournals.org
Background and Purpose—Intravascular delivery of neural stem cells (NSCs) after stroke
has been limited by the low efficiency of transendothelial migration. Vascular cell adhesion …
has been limited by the low efficiency of transendothelial migration. Vascular cell adhesion …
Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease
MA Rafi, HZ Rao, P Luzi, MT Curtis, DA Wenger - Molecular Therapy, 2012 - cell.com
Globoid cell leukodystrophy (GLD) or Krabbe disease is a neurodegenerative disorder
caused by the deficiency of the lysosomal enzyme galactocerebrosidase (GALC). This …
caused by the deficiency of the lysosomal enzyme galactocerebrosidase (GALC). This …
Intraspinal delivery of neurotrophin-3 using neural stem cells genetically modified by recombinant retrovirus
Y Liu, BT Himes, J Solowska, J Moul, SY Chow… - Experimental …, 1999 - Elsevier
Neural stem cells have been shown to participate in the repair of experimental CNS
disorders. To examine their potential in spinal cord repair, we used retroviral vectors to …
disorders. To examine their potential in spinal cord repair, we used retroviral vectors to …
Long-term improvements in lifespan and pathology in CNS and PNS after BMT plus one intravenous injection of AAVrh10-GALC in Twitcher mice
MA Rafi, HZ Rao, P Luzi, DA Wenger - Molecular Therapy, 2015 - cell.com
Krabbe disease is an autosomal recessive disorder resulting from defects in the lysosomal
enzyme galactocerebrosidase (GALC). GALC deficiency leads to severe neurological …
enzyme galactocerebrosidase (GALC). GALC deficiency leads to severe neurological …
Neural stem cells: from cell biology to cell replacement
RJE Armstrong, CN Svendsen - Cell Transplantation, 2000 - journals.sagepub.com
A large number of crippling neurological conditions result from the loss of certain cell
populations from the nervous system through disease or injury, and these cells are not …
populations from the nervous system through disease or injury, and these cells are not …
K rabbe disease: One Hundred years from the bedside to the bench to the bedside
DA Wenger, MA Rafi, P Luzi - Journal of neuroscience …, 2016 - Wiley Online Library
This Review summarizes the progress in understanding the pathogenesis and treatment of
Krabbe disease from the description of five patients in by Knud Krabbe until 2016. To …
Krabbe disease from the description of five patients in by Knud Krabbe until 2016. To …
Pre-clinical mouse models of neurodegenerative lysosomal storage diseases
There are over 50 lysosomal hydrolase deficiencies, many of which cause
neurodegeneration, cognitive decline and death. In recent years, a number of broad …
neurodegeneration, cognitive decline and death. In recent years, a number of broad …