Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis. - Wikidata - awgldk - TriplyDB

Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis.

Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis.

http://www.wikidata.org/entity/Q37463967

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Adeno Associated Viral Vector Delivered RNAi for Gene Therapy of SOD1 Amyotrophic Lateral Sclerosis RNA-targeted Therapeutics for ALS Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model.Mutant TDP-43 within motor neurons drives disease onset but not progression in amyotrophic lateral sclerosis.State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications.SOD1 silencing in motoneurons or glia rescues neuromuscular function in ALS mice.Systemic RNAi delivery to the muscles of ROSA26 mice reduces lacZ expression Initial gene vector dosing for studying symptomatology of amyotrophic lateral sclerosis in non-human primates.Gene therapy for the nervous system: challenges and new strategies.Methods for gene transfer to the central nervous system.In vivo kinetic approach reveals slow SOD1 turnover in the CNS.A Single Injection of Recombinant Adeno-Associated Virus into the Lumbar Cistern Delivers Transgene Expression Throughout the Whole Spinal Cord.Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain Developments in treatments for amyotrophic lateral sclerosis via intracerebroventricular or intrathecal delivery.Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1.Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.High content analysis in amyotrophic lateral sclerosis.Adeno-Associated Virus (AAV) as a Vector for Gene Therapy.A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model.Improved gene delivery to adult mouse spinal cord through the use of engineered hybrid adeno-associated viral serotypes.Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease.AAVrh.10 immunogenicity in mice and humans. Relevance of antibody cross-reactivity in human gene therapy.Slow intrathecal injection of rAAVrh10 enhances its transduction of spinal cord and therapeutic efficacy in a mutant SOD1 model of ALS.Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats.Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis.Streamlined ex vivo and in vivo genome editing in mouse embryos using recombinant adeno-associated viruses.AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials.Slow Infusion of Recombinant Adeno-Associated Viruses into the Mouse Cerebrospinal Fluid Space.RNA-Targeted Therapies and Amyotrophic Lateral Sclerosis.

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Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis.

http://www.wikidata.org/entity/Q37463967

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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 18 September 2013

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vedecký článok

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vetenskaplig artikel

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videnskabelig artikel

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vědecký článek

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name

Widespread spinal cord transdu ...... amyotrophic lateral sclerosis.

@en

Widespread spinal cord transdu ...... amyotrophic lateral sclerosis.

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type

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Widespread spinal cord transdu ...... amyotrophic lateral sclerosis.

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Widespread spinal cord transdu ...... amyotrophic lateral sclerosis.

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Widespread spinal cord transdu ...... amyotrophic lateral sclerosis.

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Widespread spinal cord transdu ...... amyotrophic lateral sclerosis.

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Human Molecular Genetics

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Widespread spinal cord transdu ...... amyotrophic lateral sclerosis.

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Bin Yang

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Chunxing Yang

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Guangping Gao

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Hongyan Wang

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Joshua Kramer

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Linghua Qiu

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Qin Su

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Yansu Guo

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Zuoshang Xu

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P2860

A paravascular pathway facilitates CSF flow through the brain parenchyma and the clearance of interstitial solutes, including amyloid β.

Non-cell autonomous effect of glia on motor neurons in an embryonic stem cell-based ALS model

Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons

Motor neuron degeneration in mice that express a human Cu,Zn superoxide dismutase mutation

AAV2 gene therapy readministration in three adults with congenital blindness.

Aspects of PET imaging relevant to the assessment of striatal transplantation in Huntington's disease.

Glial cell line-derived neurotrophic factor concentration dependently improves disability and motor activity in MPTP-treated common marmosets.

Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons.

Nerve injection of viral vectors efficiently transfers transgenes into motor neurons and delivers RNAi therapy against ALS.

Oligodendroglia metabolically support axons and contribute to neurodegeneration.

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668-681

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scholarly article

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10.1093/HMG/DDT454

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3

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257598908

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24108104

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amyotrophic lateral sclerosis

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3888258

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