[图书][B] Neural Stem Cell Compartments in a Mouse Model of Globoid Cell Leukodystrophy: Implication for Therapeutic Strategies

S Santambrogio - 2011 - search.proquest.com
Globoid cell leukodystrophy (GLD) is a rare genetic lysosomal disorder due to deficiency in
the B-galactocerebrosidase (GALC) enzyme. GLD affects mainly children; the prognosis is …

[HTML][HTML] Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient-and cell type-specific disease phenotypes

E Mangiameli, A Cecchele, F Morena, F Sanvito… - Stem Cell Reports, 2021 - cell.com
Globoid cell leukodystrophy (GLD) is a rare neurodegenerative lysosomal storage disease
caused by an inherited deficiency of β-galactocerebrosidase (GALC). GLD pathogenesis …

Biochemical and pathological evaluation of long-lived mice with globoid cell leukodystrophy after bone marrow transplantation

P Luzi, MA Rafi, M Zaka, HZ Rao, M Curtis… - Molecular genetics and …, 2005 - Elsevier
Globoid cell leukodystrophy (GLD) is a disorder of the central and peripheral nervous
systems caused by the deficiency of the lysosomal enzyme galactocerebrosidase (GALC) …

[PDF][PDF] Elisabeth Mangiameli, Anna Cecchele, Francesco Morena, 2 Francesca Sanvito, 3 Vittoria Matafora, 4 Angela Cattaneo, 4 Lucrezia della Volpe, Daniela Gnani …

S Martino, R Di Micco, A Bachi, A Gritti - iris.unisr.it
Globoid cell leukodystrophy (GLD) is a rare neurodegenerative lysosomal storage disease
caused by an inherited deficiency of b-galactocerebrosidase (GALC). GLD pathogenesis …

Cellular transplant therapies for globoid cell leukodystrophy: preclinical and clinical observations

KR Maher, AM Yeager - Journal of neuroscience research, 2016 - Wiley Online Library
Globoid cell leukodystrophy (GLD) is a progressive neurodegenerative disorder caused by
the deficiency of galactocerebrosidase (GALC), resulting in accumulation of toxic …

Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy

JA Hawkins-Salsbury, L Shea, X Jiang… - Journal of …, 2015 - Soc Neuroscience
Globoid cell leukodystrophy (GLD, Krabbe disease) is a lysosomal storage disease (LSD)
caused by a deficiency in galactocerebrosidase (GALC) activity. In the absence of GALC …

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 …

Experimental therapies in the murine model of globoid cell leukodystrophy

Y Li, MS Sands - Pediatric neurology, 2014 - Elsevier
Background Globoid cell leukodystrophy or Krabbe disease, is a rapidly progressive
childhood lysosomal storage disorder caused by a deficiency in galactocerebrosidase …

Meninges as an overlooked pharmacological target for Globoid Cell Leukodystrophy

A Amenta, Z Malik, Q Chiara, G Pruonto, R Alessandra… - 2023 - air.unimi.it
Abstract Globoid cell Leukodystrophy Disease (GLD) is a neurodegenerative life-threatening
disease caused by genetic defects in the lysosomal enzyme galactosylceramidase (GALC) …

Multipotent stromal cells alleviate inflammation, neuropathology, and symptoms associated with globoid cell leukodystrophy in the twitcher mouse

BA Scruggs, X Zhang, AC Bowles, PA Gold… - Stem Cells, 2013 - academic.oup.com
Globoid cell leukodystrophy (GLD) is a common neurodegenerative lysosomal storage
disorder caused by a deficiency in galactocerebrosidase (GALC), an enzyme that cleaves …