Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector. (Q45408947)

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23 December 2019

title

Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector (English)

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23 December 2019

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Jansky–Bielschowsky disease

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7

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23 December 2019

author name string

Dolan Sondhi

1

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23 December 2019

Neil R Hackett

2

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23 December 2019

Daniel A Peterson

3

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23 December 2019

Jamie Stratton

4

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23 December 2019

Michael Baad

5

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23 December 2019

Kelly M Travis

6

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23 December 2019

Ronald G Crystal

8

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23 December 2019

publication date

19 December 2006

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23 December 2019

number of pages

11

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inferred from page(s)

published in

Molecular Therapy

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23 December 2019

volume

15

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23 December 2019

issue

3

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23 December 2019

page(s)

481-491

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The neuronal ceroid-lipofuscinoses

23 December 2019

cites work

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Convection-enhanced delivery of AAV-2 combined with heparin increases TK gene transfer in the rat brain.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Immunological aspects of recombinant adeno-associated virus delivery to the mammalian brain

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Prevention of neuropathology in the mouse model of Hurler syndrome

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Efficient neuronal gene transfer with AAV8 leads to neurotoxic levels of tau or green fluorescent proteins

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Targeted retrograde gene delivery for neuronal protection

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Safety of direct administration of AAV2(CU)hCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: Implications for gene therapy using AAV vectors

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Intrapleural administration of a serotype 5 adeno-associated virus coding for alpha1-antitrypsin mediates persistent, high lung and serum levels of alpha1-antitrypsin

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Central neuronal loss and behavioral impairment in mice lacking neurotrophin receptor p75.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Distribution of tripeptidyl peptidase I in human tissues under normal and pathological conditions

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Use of quantitative TaqMan real-time PCR to track the time-dependent distribution of gene transfer vectors in vivo

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300049

7 January 2021

Identifiers

DOI

10.1038/SJ.MT.6300049

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:17180118%20AND%20SRC:MED&resulttype=core&format=json

23 December 2019

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