AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival - Wikidata - wikimedia - TriplyDB

AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival

AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival

http://www.wikidata.org/entity/Q33728295

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Clarifying lysosomal storage diseases Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan.Long-distance axonal transport of AAV9 is driven by dynein and kinesin-2 and is trafficked in a highly motile Rab7-positive compartment.Sustained normalization of neurological disease after intracranial gene therapy in a feline model.Lysosomal multienzyme complex: pros and cons of working together Myelin abnormalities in the optic and sciatic nerves in mice with GM1-gangliosidosis Gene therapy for the nervous system: challenges and new strategies.Bis(monoacylglycero)phosphate: a secondary storage lipid in the gangliosidoses.AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system.A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB.Genetic therapy for the nervous system.Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases.Progress in gene therapy for neurological disorders.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy.Enzyme replacement for GM1-gangliosidosis: Uptake, lysosomal activation, and cellular disease correction using a novel β-galactosidase:RTB lectin fusion.Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model.Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.Monosialoganglioside 1 may alleviate neurotoxicity induced by propofol combined with remifentanil in neural stem cells.Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease.Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates.Delivering drugs to the central nervous system: an overview.

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P2860

AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival

http://www.wikidata.org/entity/Q33728295

description

2010 nî lūn-bûn

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2010 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած

@hyw

2010 թվականի հոտեմբերին հրատարակված գիտական հոդված

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2010年の論文

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2010年論文

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2010年論文

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2010年論文

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2010年論文

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2010年論文

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2010年论文

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AAV-mediated gene delivery in ...... e in CNS and improves survival

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Alessandra d'Azzo

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Laryssa A Tierney

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Marike L D Broekman

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Michael A Sandberg

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Miguel Sena-Esteves

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Rena C Baek

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Stanley G Leroy

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P2860

Quantitative real-time PCR protocol for analysis of nuclear receptor signaling pathways.

A simple method for the isolation and purification of total lipides from animal tissues

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

Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid

Dysmyelinogenesis in animal model of GM1 gangliosidosis.

Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease.

AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method

Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse.

Gene therapy for lysosomal storage diseases.

Efficient gene therapy-based method for the delivery of therapeutics to primate cortex.

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Thomas N Seyfried

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2010-10-18T00:00:00Z

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47545492

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