Effective gene therapy in an authentic model of Tay-Sachs-related diseases - Wikidata - thesis-toulmin - TriplyDB

Effective gene therapy in an authentic model of Tay-Sachs-related diseases

Effective gene therapy in an authentic model of Tay-Sachs-related diseases

http://www.wikidata.org/entity/Q30477455

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Therapeutic potential of intracerebroventricular replacement of modified human β-hexosaminidase B for GM2 gangliosidosis Clarifying lysosomal storage diseases Animal models of GM2 gangliosidosis: utility and limitations Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates Characterization of inducible models of Tay-Sachs and related disease Natural history of infantile G(M2) gangliosidosis.Pathology of GM2 gangliosidosis in Jacob sheep Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease Gene transfer corrects acute GM2 gangliosidosis--potential therapeutic contribution of perivascular enzyme flow Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.Sustained normalization of neurological disease after intracranial gene therapy in a feline model.Imaging MALDI mass spectrometry of sphingolipids using an oscillating capillary nebulizer matrix application system.AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival Lyso-GM2 ganglioside: a possible biomarker of Tay-Sachs disease and Sandhoff disease Insights into the evolutionary features of human neurodegenerative diseases.In cellulo examination of a beta-alpha hybrid construct of beta-hexosaminidase A subunits, reported to interact with the GM2 activator protein and hydrolyze GM2 ganglioside AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system.Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10 Comparative analysis of brain lipids in mice, cats, and humans with Sandhoff disease Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders.Substrate reduction therapy.Reversibility of neuropathology in Tay-Sachs-related diseases.Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease.Clinical neurogenetics: neuropathic lysosomal storage disorders.Secondary alterations of sphingolipid metabolism in lysosomal storage diseases.Newborn screening for lysosomal storage disorders.Lysosomal lipid storage diseases.The cellular pathology of lysosomal diseases.Gene therapy for the neurological manifestations in lysosomal storage disorders.Sphingolipid lysosomal storage disorders.Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo.Viral vectors for therapy of neurologic diseases.Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.Personalizing Stem Cell Research and Therapy: The Arduous Road Ahead or Missed Opportunity?Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease.Sphingolipid signalling and liver diseases.Β-hexosaminidase over-expression affects lysosomal glycohydrolases expression and glycosphingolipid metabolism in mammalian cells.Early deficits in motor coordination and cognitive dysfunction in a mouse model of the neurodegenerative lysosomal storage disorder, Sandhoff disease.Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain.Haematopoietic Stem Cell Transplantation Arrests the Progression of Neurodegenerative Disease in Late-Onset Tay-Sachs Disease.

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Effective gene therapy in an authentic model of Tay-Sachs-related diseases

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2006 nî lūn-bûn

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2006年の論文

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Effective gene therapy in an authentic model of Tay-Sachs-related diseases

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Proceedings of the National Academy of Sciences of the United States of America

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Effective gene therapy in an authentic model of Tay-Sachs-related diseases

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M 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

Use of avidin-biotin-peroxidase complex (ABC) in immunoperoxidase techniques: a comparison between ABC and unlabeled antibody (PAP) procedures

The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement

Deletion of macrophage-inflammatory protein 1 alpha retards neurodegeneration in Sandhoff disease mice

Mouse models of Tay-Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism

Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column.

Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors.

Possible role of autoantibodies in the pathophysiology of GM2 gangliosidoses.

GM2 ganglioside lysosomal storage disease in cats with beta-hexosaminidase deficiency.

Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin.

Microglial activation precedes acute neurodegeneration in Sandhoff disease and is suppressed by bone marrow transplantation.

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