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

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

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

http://www.wikidata.org/entity/Q33713769

about

Telomerase gene therapy in adult and old mice delays aging and increases longevity without increasing cancer Rett Syndrome: Crossing the Threshold to Clinical Translation 5p deletions: Current knowledge and future directions.State-of-the-art human gene therapy: part I. Gene delivery technologies Perinatal systemic gene delivery using adeno-associated viral vectors Spinal muscular atrophy: development and implementation of potential treatments Current prospects and challenges for epilepsy gene therapy Noninvasive and targeted gene delivery into the brain using microbubble-facilitated focused ultrasound.Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model.Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9 Applications of CRISPR/Cas9 for Gene Editing in Hereditary Movement Disorders Drug and gene delivery across the blood-brain barrier with focused ultrasound BAAV transcytosis requires an interaction with beta-1-4 linked- glucosamine and gp96 Targeted gene transfer to the brain via the delivery of brain-penetrating DNA nanoparticles with focused ultrasound.Gene delivery to the spinal cord using MRI-guided focused ultrasound Pathogenesis of developmental anomalies of the central nervous system induced by congenital cytomegalovirus infection.Targeted delivery of self-complementary adeno-associated virus serotype 9 to the brain, using magnetic resonance imaging-guided focused ultrasound CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy.Expansive gene transfer in the rat CNS rapidly produces amyotrophic lateral sclerosis relevant sequelae when TDP-43 is overexpressed.Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder.Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy Requirement of enhanced Survival Motoneuron protein imposed during neuromuscular junction maturation.Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB).Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9.Systemic injection of AAV9-GDNF provides modest functional improvements in the SOD1G93A ALS rat but has adverse side effects.Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment Systemic gene delivery to the central nervous system using Adeno-associated virus.Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10 Spinal muscular atrophy: mechanisms and therapeutic strategies.Gene delivery with viral vectors for cerebrovascular diseases.Defining the therapeutic window in a severe animal model of spinal muscular atrophy Peptide ligands incorporated into the threefold spike capsid domain to re-direct gene transduction of AAV8 and AAV9 in vivo Over the barrier and through the blood: to CNS delivery we go.Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.Temporal requirement for high SMN expression in SMA mice Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders SMN-inducing compounds for the treatment of spinal muscular atrophy.

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P2860

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

http://www.wikidata.org/entity/Q33713769

description

2009 nî lūn-bûn

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2009 թուականի Ապրիլին հրատարակուած գիտական յօդուած

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2009 թվականի ապրիլին հրատարակված գիտական հոդված

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2009年の論文

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Molecular Therapy

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Intravenous administration of ...... livery to adult motor neurons.

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Anne-Marie Douar

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Béatrice Joussemet

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Christel Riviere

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John Fyfe

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Laurence Dubreil

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Marie-Anne Colle

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Martine Barkats

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Philippe Moullier

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Sandra Duque

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Thibaut Marais

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P2860

Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND)

Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats

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

Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis

Onset and progression in inherited ALS determined by motor neurons and microglia

Targeted expression of an oncogenic adaptor protein v-Crk potentiates axonal growth in dorsal root ganglia and motor neurons in vivo.

Drug delivery to brain via the blood-brain barrier.

Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

Immunohistochemical study on choline acetyltransferase in the spinal cord of patients with amyotrophic lateral sclerosis.

Self-complementary AAV vectors; advances and applications.

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1187-1196

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10.1038/MT.2009.71

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7

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17

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19367261

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