Treatment for Krabbe's disease: Finding the combination

CR Mikulka, MS Sands - Journal of neuroscience research, 2016 - Wiley Online Library
Globoid cell leukodystrophy (GLD) is an autosomal recessive neurodegenerative disorder
caused by a deficiency of the lysosomal enzyme galactocerebrosidase (GALC). GALC is …

Spheroid mesenchymal stem cells and mesenchymal stem cell-derived microvesicles: two potential therapeutic strategies

L Xie, M Mao, L Zhou, B Jiang - Stem Cells and Development, 2016 - liebertpub.com
Mesenchymal stem cells (MSCs) have drawn worldwide attention of scientists and clinicians
due to their ability to differentiate into other cell lineages, secrete paracrine factors, modulate …

Lysosomal re-acidification prevents lysosphingolipid-induced lysosomal impairment and cellular toxicity

CJ Folts, N Scott-Hewitt, C Pröschel… - PLoS …, 2016 - journals.plos.org
Neurodegenerative lysosomal storage disorders (LSDs) are severe and untreatable, and
mechanisms underlying cellular dysfunction are poorly understood. We found that toxic …

CNS axons globally increase axonal transport after peripheral conditioning

FM Mar, AR Simoes, S Leite, MM Morgado… - Journal of …, 2014 - Soc Neuroscience
Despite the inability of CNS axons to regenerate, an increased regenerative capacity can be
elicited following conditioning lesion to the peripheral branch of dorsal root ganglia neurons …

Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy

A Ricca, N Rufo, S Ungari, F Morena… - Human molecular …, 2015 - academic.oup.com
Globoid cell leukodystrophy (GLD) is a lysosomal storage disease caused by deficient
activity of β-galactocerebrosidase (GALC). The infantile forms manifest with rapid and …

Early axonal loss accompanied by impaired endocytosis, abnormal axonal transport, and decreased microtubule stability occur in the model of Krabbe's disease

CA Teixeira, CO Miranda, VF Sousa, TE Santos… - Neurobiology of …, 2014 - Elsevier
In Krabbe's disease (KD), a leukodystrophy caused by β-galactosylceramidase deficiency,
demyelination and a myelin-independent axonopathy contributes to the severe …

The dyslexia-susceptibility protein KIAA0319 inhibits axon growth through Smad2 signaling

F Franquinho, J Nogueira-Rodrigues… - Cerebral …, 2017 - academic.oup.com
KIAA0319 is a transmembrane protein associated with dyslexia with a presumed role in
neuronal migration. Here we show that KIAA0319 expression is not restricted to the brain but …

The miR-210 primed endothelial progenitor cell exosomes alleviate acute ischemic brain injury

J Wang, S Chen, H Sawant, Y Chen… - Current Stem Cell …, 2024 - benthamdirect.com
Background: Stem cell-released exosomes (EXs) have shown beneficial effects on
regenerative diseases. Our previous study has revealed that EXs of endothelial progenitor …

Repeated mesenchymal stromal cell treatment sustainably alleviates Machado-Joseph disease

CO Miranda, A Marcelo, TP Silva, J Barata… - Molecular Therapy, 2018 - cell.com
Machado-Joseph disease (MJD) or spinocerebellar ataxia type 3, the most common
dominant spinocerebellar ataxia (SCA) worldwide, is caused by over-repetition of a CAG …

Adult bone marrow: which stem cells for cellular therapy protocols in neurodegenerative disorders?

S Wislet-Gendebien, E Laudet… - BioMed Research …, 2012 - Wiley Online Library
The generation of neuronal cells from stem cells obtained from adult bone marrow is of
significant clinical interest in order to design new cell therapy protocols for several …