Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. - Wikidata - wikimedia - TriplyDB

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

http://www.wikidata.org/entity/Q34778098

about

Manufacturing of recombinant adeno-associated viral vectors for clinical trials Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan.Noninvasive and targeted gene delivery into the brain using microbubble-facilitated focused ultrasound.Exosome-associated AAV vector as a robust and convenient neuroscience tool AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease.Crystal Structure and Autoactivation Pathway of the Precursor Form of Human Tripeptidyl-peptidase 1, the Enzyme Deficient in Late Infantile Ceroid Lipofuscinosis A critical tryptophan and Ca2+ in activation and catalysis of TPPI, the enzyme deficient in classic late-infantile neuronal ceroid lipofuscinosis Gene therapy for metachromatic leukodystrophy.Reversal of depressed behaviors in mice by p11 gene therapy in the nucleus accumbens.Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice.Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy Long-distance axonal transport of AAV9 is driven by dynein and kinesin-2 and is trafficked in a highly motile Rab7-positive compartment.Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene.Sustained normalization of neurological disease after intracranial gene therapy in a feline model.Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy.Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy Striatal readministration of rAAV vectors reveals an immune response against AAV2 capsids that can be circumvented.Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders.AAV provides an alternative for gene therapy of the peripheral sensory nervous system.Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions.Gene therapy in epilepsy.Gene transfer to the CNS using recombinant adeno-associated virus Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes.Therapeutic approaches for lysosomal storage diseases Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease).High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines.Beyond the hammer and the scalpel: selective circuit control for the epilepsies Gene therapy for lysosomal storage disorders: a good start.p11 and gene therapy for severe psychiatric disorders: a practical goal?Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial.Capsid serotype and timing of injection determines AAV transduction in the neonatal mice brain.Genetic modification of neurons to express bevacizumab for local anti-angiogenesis treatment of glioblastoma.Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases Dipeptidyl-peptidase I does not functionally compensate for the loss of tripeptidyl-peptidase I in the neurodegenerative disease late-infantile neuronal ceroid lipofuscinosis.Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease.When ethics constrains clinical research: trial design of control arms in "greater than minimal risk" pediatric trials Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis.

P2860

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P2860

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

http://www.wikidata.org/entity/Q34778098

description

2008 nî lūn-bûn

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2008 թուականի Մայիսին հրատարակուած գիտական յօդուած

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2008 թվականի մայիսին հրատարակված գիտական հոդված

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2008年の論文

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Treatment of late infantile ne ...... ed virus expressing CLN2 cDNA.

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Treatment of late infantile ne ...... ed virus expressing CLN2 cDNA.

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Treatment of late infantile ne ...... ed virus expressing CLN2 cDNA.

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Treatment of late infantile ne ...... ed virus expressing CLN2 cDNA.

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Human Gene Therapy

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Treatment of late infantile ne ...... ed virus expressing CLN2 cDNA.

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Barry Kosofsky

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Bruce M Greenwald

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Dolan Sondhi

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Douglas Ballon

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Jonathan P Dyke

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Linda Heier

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Madhu Mazumdar

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Michael G Kaplitt

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Minal V Kekatpure

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10.1089/HUM.2008.022

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Infantile neuronal ceroid lipofuscinosis