(Q45885520)

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AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL.

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4 January 2020

AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL (English)

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4 January 2020

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Stephen M. Kaminsky

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4 January 2020

Kimberly B Bjugstad

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4 January 2020

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Sondhi D

1

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4 January 2020

Peterson DA

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4 January 2020

Giannaris EL

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Sanders CT

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Mendez BS

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4 January 2020

De B

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4 January 2020

Rostkowski AB

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Blanchard B

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Sladek JR Jr

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4 January 2020

Redmond DE Jr

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4 January 2020

Leopold PL

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4 January 2020

Hackett NR

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4 January 2020

Crystal RG

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4 January 2020

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1 November 2005

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4 January 2020

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4 January 2020

12

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4 January 2020

22

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4 January 2020

1618-1632

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4 January 2020

https://scigraph.springernature.com/pub.10.1038/sj.gt.3302549

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The neuronal ceroid-lipofuscinoses

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Association of mutations in a lysosomal protein with classical late-infantile neuronal ceroid lipofuscinosis

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Classical late infantile neuronal ceroid lipofuscinosis fibroblasts are deficient in lysosomal tripeptidyl peptidase I

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A lysosomal proteinase, the late infantile neuronal ceroid lipofuscinosis gene (CLN2) product, is essential for degradation of a hydrophobic protein, the subunit c of ATP synthase.

1 reference

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Retinal degeneration in retinitis pigmentosa and neuronal ceroid lipofuscinosis: An overview

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Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain

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Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector

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Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

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21 January 2018

Viral-mediated delivery of the late-infantile neuronal ceroid lipofuscinosis gene, TPP-I to the mouse central nervous system

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21 January 2018

Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice.

1 reference

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21 January 2018

Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors.

1 reference

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21 January 2018

Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse

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The human CLN2 protein/tripeptidyl-peptidase I is a serine protease that autoactivates at acidic pH

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21 January 2018

Mutational analysis of the defective protease in classic late-infantile neuronal ceroid lipofuscinosis, a neurodegenerative lysosomal storage disorder

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21 January 2018

A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration

1 reference

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21 January 2018

Adeno-associated virus-mediated gene transfer to the brain: duration and modulation of expression

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21 January 2018

Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors

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21 January 2018

Feasibility of gene therapy for late neuronal ceroid lipofuscinosis

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Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

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21 January 2018

Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector

1 reference

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21 January 2018

Dose and promoter effects of adeno-associated viral vector for green fluorescent protein expression in the rat brain.

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Dopaminergic cell loss induced by human A30P alpha-synuclein gene transfer to the rat substantia nigra

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Inactivation of the very strong HCMV immediate early promoter by DNA CpG methylation in vitro

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Promoters and regulatory elements that improve adeno-associated virus transgene expression in the brain

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Organization of the thalamostriatal projections in the rat, with special emphasis on the ventral striatum

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Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

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Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

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Tripeptidyl peptidase I, the late infantile neuronal ceroid lipofuscinosis gene product, initiates the lysosomal degradation of subunit c of ATP synthase

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Characterization of intrastriatal recombinant adeno-associated virus-mediated gene transfer of human tyrosine hydroxylase and human GTP-cyclohydrolase I in a rat model of Parkinson's disease.

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Identifiers

10.1038/SJ.GT.3302549

1 reference

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4 January 2020

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4 January 2020

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