Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis.
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Moving towards effective therapeutic strategies for Neuronal Ceroid LipofuscinosisNeuroanatomy goes viral!Gene therapy for metachromatic leukodystrophy.Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders.Intra-arterial delivery of AAV vectors to the mouse brain after mannitol mediated blood brain barrier disruptionGenetic modification of neurons to express bevacizumab for local anti-angiogenesis treatment of glioblastoma.Human serum albumin-based nanoparticle-mediated in vitro gene deliveryGene therapy for the nervous system: challenges and new strategies.Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho(-/-) mouseClinical applications involving CNS gene transfer.Adenoviral and adeno-associated viral vectors-mediated neuronal gene transfer to cardiovascular control regions of the rat brainHumoral Immune Response to AAV.Radioiodinated Capsids Facilitate In Vivo Non-Invasive Tracking of Adeno-Associated Gene Transfer Vectors.Role of endosomes and lysosomes in human disease.Adeno-associated virus vectors and neurological gene therapy.Preclinical toxicity evaluation of AAV for pain: evidence from human AAV studies and from the pharmacology of analgesic drugs.Future perspectives: Moving towards NCL treatments.Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.C3 transferase gene therapy for continuous conditional RhoA inhibition.Intraneural convection enhanced delivery of AAVrh20 for targeting primary sensory neurons.Non-invasive gene targeting to the fetal brain after intravenous administration and transplacental transfer of plasmid DNA using PEGylated immunoliposomes.Toward a gene therapy for neurological and somatic MPSIIIAAAVrh.10-mediated APOE2 CNS Gene Therapy for APOE4-associated Alzheimer's Disease.Recent progress and considerations for AAV gene therapies targeting the central nervous system.Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten DiseaseAdeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer
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P2860
Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis.
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
@pt
bilimsel makale
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scientific article published on October 2012
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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
Long-term expression and safet ...... euronal ceroid lipofuscinosis.
@en
Long-term expression and safet ...... euronal ceroid lipofuscinosis.
@nl
type
label
Long-term expression and safet ...... euronal ceroid lipofuscinosis.
@en
Long-term expression and safet ...... euronal ceroid lipofuscinosis.
@nl
prefLabel
Long-term expression and safet ...... euronal ceroid lipofuscinosis.
@en
Long-term expression and safet ...... euronal ceroid lipofuscinosis.
@nl
P2093
P2860
P50
P356
P1476
Long-term expression and safet ...... neuronal ceroid lipofuscinosis
@en
P2093
Barry Kosofsky
Dolan Sondhi
Keith Purpura
Linda Johnson
Mark M Souweidane
Michael G Kaplitt
Neil R Hackett
Ronald G Crystal
Sebastien Monette
Stephen M Kaminksy
P2860
P304
P356
10.1089/HGTB.2012.120
P577
2012-10-01T00:00:00Z