Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates. - Wikidata - wikimedia - TriplyDB

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates.

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates.

http://www.wikidata.org/entity/Q36745664

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Biology of adeno-associated viral vectors in the central nervous system Spinal muscular atrophy: development and implementation of potential treatments 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)A large animal model of spinal muscular atrophy and correction of phenotype.E Pluribus Unum: 50 Years of Research, Millions of Viruses, and One Goal--Tailored Acceleration of AAV Evolution Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.Sustained normalization of neurological disease after intracranial gene therapy in a feline model.Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery.Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-α-L-Iduronidase for Hurler Disease Supraspinal gene transfer by intrathecal adeno-associated virus serotype 5.Chondroitin Sulfate is the Primary Receptor for a Peptide-Modified AAV That Targets Brain Vascular Endothelium In Vivo.Intracisternal delivery of NFκB-inducible scAAV2/9 reveals locoregional neuroinflammation induced by systemic kainic acid treatment.Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.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.Widespread and efficient transduction of spinal cord and brain following neonatal AAV injection and potential disease modifying effect in ALS mice.Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase.Current gene therapy using viral vectors for chronic pain Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna.Making the White Matter Matters: Progress in Understanding Canavan's Disease and Therapeutic Interventions Through Eight Decades.Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids.Clinical applications involving CNS gene transfer.Methods for gene transfer to the central nervous system.A numerical investigation of intrathecal isobaric drug dispersion within the cervical subarachnoid space 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.Gene Therapy: The View from NCATS.AAV9-mediated central nervous system-targeted gene delivery via cisterna magna route in mice.Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10 Targeted axonal import (TAxI) peptide delivers functional proteins into spinal cord motor neurons after peripheral administration.A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs.Recent advances in RNA interference therapeutics for CNS diseases.MicroRNA as therapeutic targets for treatment of depression.Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant Minimally invasive convection-enhanced delivery of biologics into dorsal root ganglia: validation in the pig model and prospective modeling in humans. Technical note.

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Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates.

http://www.wikidata.org/entity/Q36745664

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Global CNS gene delivery and e ...... tration in non-human primates.

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R Jude Samulski

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S J Gray

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S Nagabhushan Kalburgi

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T J McCown

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P2860

Drug transport in brain via the cerebrospinal fluid

Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus

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

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB).

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

Differential adeno-associated virus mediated gene transfer to sensory neurons following intrathecal delivery by direct lumbar puncture.

Intrathecal delivery of a mutant micro-opioid receptor activated by naloxone as a possible antinociceptive paradigm.

Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.

Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

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10.1038/GT.2012.101

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