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 …
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 …
[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 …
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) …
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 …
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 …
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
M Sessa, L Lorioli, F Fumagalli, S Acquati, D Redaelli… - The Lancet, 2016 - thelancet.com
Background Metachromatic leukodystrophy (a deficiency of arylsulfatase A [ARSA]) is a fatal
demyelinating lysosomal disease with no approved treatment. We aimed to assess the long …
demyelinating lysosomal disease with no approved treatment. We aimed to assess the long …
[HTML][HTML] Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase …
Background Effective treatment for metachromatic leukodystrophy (MLD) remains a
substantial unmet medical need. In this study we investigated the safety and efficacy of …
substantial unmet medical need. In this study we investigated the safety and efficacy of …
Gene therapy for metachromatic leukodystrophy
JB Rosenberg, SM Kaminsky… - Journal of …, 2016 - Wiley Online Library
Leukodystrophies (LDs) are rare, often devastating genetic disorders with neurologic
symptoms. There are currently no disease‐specific therapeutic approaches for these …
symptoms. There are currently no disease‐specific therapeutic approaches for these …
Enzyme, cell and gene‐based therapies for metachromatic leukodystrophy
C Sevin, P Aubourg, N Cartier - … Journal of the Society for the …, 2007 - Wiley Online Library
Metachromatic leukodystrophy (MLD) is a demyelinating storage disease caused by
deficiency of the lysosomal enzyme arylsulfatase A (ARSA). Lack of ARSA activity leads to …
deficiency of the lysosomal enzyme arylsulfatase A (ARSA). Lack of ARSA activity leads to …
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