Reversibility of neuropathology in Tay-Sachs-related diseases. - Wikidata - awgldk - TriplyDB

Reversibility of neuropathology in Tay-Sachs-related diseases.

Reversibility of neuropathology in Tay-Sachs-related diseases.

http://www.wikidata.org/entity/Q37463989

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Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy.In Vivo NMR Studies of the Brain with Hereditary or Acquired Metabolic Disorders.AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system.Protease-resistant modified human β-hexosaminidase B ameliorates symptoms in GM2 gangliosidosis model Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease.The progressive myoclonic epilepsies.Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo.AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff disease.Combined replacement effects of human modified β-hexosaminidase B and GM2 activator protein on GM2 gangliosidoses fibroblasts.A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells.

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Reversibility of neuropathology in Tay-Sachs-related diseases.

http://www.wikidata.org/entity/Q37463989

description

article científic

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article scientifique

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articolo scientifico

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artigo científico

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bilimsel makale

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scientific article published on 20 September 2013

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vedecký článok

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vetenskaplig artikel

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videnskabelig artikel

@da

vědecký článek

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name

Reversibility of neuropathology in Tay-Sachs-related diseases.

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Reversibility of neuropathology in Tay-Sachs-related diseases.

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type

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Reversibility of neuropathology in Tay-Sachs-related diseases.

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Reversibility of neuropathology in Tay-Sachs-related diseases.

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Reversibility of neuropathology in Tay-Sachs-related diseases.

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Reversibility of neuropathology in Tay-Sachs-related diseases.

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Human Molecular Genetics

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Reversibility of neuropathology in Tay-Sachs-related diseases.

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María-Begoña Cachón-González

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Robin Ziegler

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Seng H Cheng

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Susan Z Wang

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Timothy M Cox

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P2860

Selective attraction of monocytes and T lymphocytes of the memory phenotype by cytokine RANTES

AB variant of infantile GM2 gangliosidosis: deficiency of a factor necessary for stimulation of hexosaminidase A-catalyzed degradation of ganglioside GM2 and glycolipid GA2

Characterization of inducible models of Tay-Sachs and related disease

alpha-Synuclein locus triplication causes Parkinson's disease

Variation of beta-N-acetylhexosaminidase-pattern in Tay-Sachs disease

A block of autophagy in lysosomal storage disorders

Deficient hexozaminidase activity in an exceptional case of Tay-Sachs disease with additional storage of kidney globoside in visceral organs

Tay-Sachs disease: generalized absence of a beta-D-N-acetylhexosaminidase component

Pathology of GM2 gangliosidosis in Jacob sheep

Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease

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scholarly article

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10.1093/HMG/DDT459

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3

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24057669

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