Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector. - Wikidata - awgldk - TriplyDB

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.

http://www.wikidata.org/entity/Q36951265

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Adeno Associated Viral Vector Delivered RNAi for Gene Therapy of SOD1 Amyotrophic Lateral Sclerosis In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy Better Targeting, Better Efficiency for Wide-Scale Neuronal Transduction with the Synapsin Promoter and AAV-PHP.B.Tailored transgene expression to specific cell types in the central nervous system after peripheral injection with AAV9.Site-Specific PEGylated Adeno-Associated Viruses with Increased Serum Stability and Reduced Immunogenicity.Viral vectors for therapy of neurologic diseases.Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Systemic delivery of adeno-associated viral vectors.Therapeutic Potential of Secreted Amyloid Precursor Protein APPsα.Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems.Brain endothelial cell-targeted gene therapy of neurovascular disorders.In vivo genome editing in animals using AAV-CRISPR system: applications to translational research of human disease.Engineered viral vectors for functional interrogation, deconvolution, and manipulation of neural circuits.Nuclease-Mediated Gene Therapies for Inherited Metabolic Diseases of the Liver.In vivo methods for acute modulation of gene expression in the central nervous system Neuro-Immuno-Gene- and Genome-Editing-Therapy for Alzheimer's Disease: Are We There Yet?Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

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P2860

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.

http://www.wikidata.org/entity/Q36951265

description

2015 nî lūn-bûn

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Widespread Central Nervous Sys ...... livery of Novel AAV-AS Vector.

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Widespread Central Nervous Sys ...... livery of Novel AAV-AS Vector.

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Widespread Central Nervous Sys ...... livery of Novel AAV-AS Vector.

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Molecular Therapy

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Widespread Central Nervous Sys ...... livery of Novel AAV-AS Vector.

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Aime K Johnson

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Allison M Keeler

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Anne F Harris

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Douglas R Martin

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Ellen Sapp

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Guangping Gao

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Heather L Gray-Edwards

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Jacob A Johnson

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Jennifer S Ferreira

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Lorelei Stoica

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P2860

Huntingtin is a cytoplasmic protein associated with vesicles in human and rat brain neurons

The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy.

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Adeno-associated virus type 2 VP2 capsid protein is nonessential and can tolerate large peptide insertions at its N terminus

Directed evolution of adeno-associated virus to an infectious respiratory virus

Analysis of Relative Gene Expression Data Using Real-Time Quantitative PCR and the 2−ΔΔCT Method

Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9

A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes.

A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.

Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington's disease

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726-735

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10.1038/MT.2015.231

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4

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24

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Damien J Cabral

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