[HTML][HTML] Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice
A Biffi, A Capotondo, S Fasano… - The Journal of …, 2006 - Am Soc Clin Investig
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by deficiency
of the enzyme arylsulfatase A (ARSA). MLD is characterized by progressive demyelination …
of the enzyme arylsulfatase A (ARSA). MLD is characterized by progressive demyelination …
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
A Biffi, E Montini, L Lorioli, M Cesani, F Fumagalli… - Science, 2013 - science.org
Introduction Metachromatic leukodystrophy (MLD) is a neurodegenerative lysosomal
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …
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) …
In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice
A Consiglio, A Quattrini, S Martino, JC Bensadoun… - Nature medicine, 2001 - nature.com
Metachromatic leukodystrophy (MLD) is a lipidosis caused by deficiency of arylsulfatase A
(ARSA). Although the genetics of MLD are known, its pathophysiology is not understood …
(ARSA). Although the genetics of MLD are known, its pathophysiology is not understood …
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 …
Correction of brain oligodendrocytes by AAVrh. 10 intracerebral gene therapy in metachromatic leukodystrophy mice
F Piguet, D Sondhi, M Piraud, F Fouquet… - Human gene …, 2012 - liebertpub.com
Metachromatic leukodystrophy (MLD) is a lysosomal storage disorder characterized by
accumulation of sulfatides in glial cells and neurons, the result of an inherited deficiency of …
accumulation of sulfatides in glial cells and neurons, the result of an inherited deficiency of …
Intracerebral gene therapy using AAVrh. 10-hARSA recombinant vector to treat patients with early-onset forms of metachromatic leukodystrophy: preclinical feasibility …
No treatment is available for early-onset forms of metachromatic leukodystrophy (MLD), a
lysosomal storage disease caused by autosomal recessive defect in arylsulfatase A (ARSA) …
lysosomal storage disease caused by autosomal recessive defect in arylsulfatase A (ARSA) …
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 …
Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age
F Matthes, S Stroobants, D Gerlach… - Human molecular …, 2012 - academic.oup.com
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by a functional
deficiency of arylsulfatase A (ASA). Previous studies in ASA-knockout mice suggested …
deficiency of arylsulfatase A (ASA). Previous studies in ASA-knockout mice suggested …
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 …
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