Mechanisms of demyelination and neurodegeneration in globoid cell leukodystrophy

ML Feltri, NI Weinstock, J Favret, N Dhimal, L Wrabetz… - Glia, 2021 - Wiley Online Library
Globoid cell leukodystrophy (GLD), also known as Krabbe disease, is a lysosomal storage
disorder causing extensive demyelination in the central and peripheral nervous systems …

[HTML][HTML] Nanomedicines to treat rare neurological disorders: The case of Krabbe disease

TL Moore, G Pannuzzo, G Costabile… - Advanced Drug Delivery …, 2023 - Elsevier
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 …

Isolation of amniotic stem cell lines with potential for therapy

P De Coppi, G Bartsch Jr, MM Siddiqui, T Xu… - Nature …, 2007 - nature.com
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 …

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 …

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 …

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 …

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 …

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 …

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 …

Pre-clinical mouse models of neurodegenerative lysosomal storage diseases

JM Favret, NI Weinstock, ML Feltri… - Frontiers in Molecular …, 2020 - frontiersin.org
There are over 50 lysosomal hydrolase deficiencies, many of which cause
neurodegeneration, cognitive decline and death. In recent years, a number of broad …