Arylsulfatase A overexpressing human iPSC-derived neural cells reduce CNS sulfatide storage in a mouse model of metachromatic leukodystrophy
Metachromatic leukodystrophy (MLD) is an inherited lysosomal storage disorder resulting
from a functional deficiency of arylsulfatase A (ARSA), an enzyme that catalyzes desulfation …
from a functional deficiency of arylsulfatase A (ARSA), an enzyme that catalyzes desulfation …
Metabolic correction in oligodendrocytes derived from metachromatic leukodystrophy mouse model by using encapsulated recombinant myoblasts
A Consiglio, S Martino, D Dolcetta, G Cusella… - Journal of the …, 2007 - Elsevier
In an effort to develop an encapsulated cell-based system to deliver arylsulfatase A (ARSA)
to the central nervous system of metachromatic leukodystrophy (MLD) patients, we …
to the central nervous system of metachromatic leukodystrophy (MLD) patients, we …
Oligodendroglial progenitor cell therapy limits central neurological deficits in mice with metachromatic leukodystrophy
MI Givogri, F Galbiati, S Fasano, S Amadio… - Journal of …, 2006 - Soc Neuroscience
This work describes the first successful oligodendrocyte-based cell therapy for
presymptomatic arylsulfatase A (ARSA) null neonate mice, a murine model for human …
presymptomatic arylsulfatase A (ARSA) null neonate mice, a murine model for human …
Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy Arsa Knock-Out Mice
TS Martin, TA Seabrook, K Gall, J Newman… - Journal of …, 2023 - Soc Neuroscience
Metachromatic leukodystrophy (MLD) is a rare, inherited, demyelinating lysosomal storage
disorder caused by mutations in the arylsulfatase-A gene (ARSA). In patients, levels of …
disorder caused by mutations in the arylsulfatase-A gene (ARSA). In patients, levels of …
Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer
C Sevin, L Verot, A Benraiss, D Van Dam, D Bonnin… - Gene therapy, 2007 - nature.com
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by genetic
deficiency of arylsulfatase A (ARSA) enzyme. Failure in catalyzing the degradation of its …
deficiency of arylsulfatase A (ARSA) enzyme. Failure in catalyzing the degradation of its …
Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy
C Sevin, A Benraiss, D Van Dam… - Human Molecular …, 2006 - academic.oup.com
Metachromatic leukodystrophy (MLD) is a neurodegenerative lysosomal disease caused by
a defect of the enzyme arylsulfatase A (ARSA) that disrupts the degradation of sulfatides …
a defect of the enzyme arylsulfatase A (ARSA) that disrupts the degradation of sulfatides …
[HTML][HTML] Complete correction of brain and spinal cord pathology in metachromatic leukodystrophy mice
E Audouard, V Oger, B Meha, N Cartier… - Frontiers in Molecular …, 2021 - frontiersin.org
Metachromatic leukodystrophy (MLD) is a lysosomal storage disorder characterized by
accumulation of sulfatides in both glial cells and neurons. MLD results from an inherited …
accumulation of sulfatides in both glial cells and neurons. MLD results from an inherited …
Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy
A Capotondo, M Cesani, S Pepe, S Fasano… - Human gene …, 2007 - liebertpub.com
Successful gene therapy approaches for metachromatic leukodystrophy (MLD), based either
on hematopoietic stem/progenitor cells (HSPCs) or direct central nervous system (CNS) …
on hematopoietic stem/progenitor cells (HSPCs) or direct central nervous system (CNS) …
Human iPSC-based models highlight defective glial and neuronal differentiation from neural progenitor cells in metachromatic leukodystrophy
The pathological cascade leading from primary storage to neural cell dysfunction and death
in metachromatic leukodystrophy (MLD) has been poorly elucidated in human-derived …
in metachromatic leukodystrophy (MLD) has been poorly elucidated in human-derived …
Increasing sulfatide synthesis in myelin-forming cells of arylsulfatase A-deficient mice causes demyelination and neurological symptoms reminiscent of human …
H Ramakrishnan, KK Hedayati… - Journal of …, 2007 - Soc Neuroscience
Metachromatic leukodystrophy (MLD) is a lysosomal storage disorder caused by the
deficiency of arylsulfatase A (ASA). This results in accumulation of sulfated …
deficiency of arylsulfatase A (ASA). This results in accumulation of sulfated …
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