(Q44039833)

29 November 2017

Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease (English)

1 reference

29 November 2017

Mario A Cabrera-Salazar

1

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29 November 2017

Eric M Roskelley

2

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29 November 2017

Jie Bu

3

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29 November 2017

Bradley L Hodges

4

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29 November 2017

Nelson Yew

5

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29 November 2017

James C Dodge

6

1 reference

29 November 2017

Lamya S Shihabuddin

7

1 reference

29 November 2017

Istvan Sohar

8

1 reference

29 November 2017

David E Sleat

9

1 reference

29 November 2017

Ronald K Scheule

10

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29 November 2017

Beverly L Davidson

11

1 reference

29 November 2017

Seng H Cheng

12

1 reference

29 November 2017

Peter Lobel

13

1 reference

29 November 2017

Marco A Passini

14

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29 November 2017

17 July 2007

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29 November 2017

1 reference

29 November 2017

15

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29 November 2017

10

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29 November 2017

1782-1788

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

29 November 2017

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Purification and characterisation of a tripeptidyl aminopeptidase I from rat spleen

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Defect of proteolysis of mitochondrial ATP synthase subunit C in neuronal ceroid lipofuscinosis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

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.6300249

7 January 2021

Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

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.6300249

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Gene therapy for lysosomal storage diseases.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-defici

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Effective gene therapy for an inherited CNS disease in a large animal model

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Complete correction of enzymatic deficiency and neurochemistry in the GM1-gangliosidosis mouse brain by neonatal adeno-associated virus-mediated gene delivery

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

AAV1 mediated co-expression of formylglycine-generating enzyme and arylsulfatase a efficiently corrects sulfatide storage in a mouse model of metachromatic leukodystrophy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Quantifiable bradykinesia, gait abnormalities and Huntington's disease-like striatal lesions in rats chronically treated with 3-nitropropionic acid

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Proactive transplantation of human neural stem cells prevents degeneration of striatal neurons in a rat model of Huntington disease

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Comparative effects of lesions to the ponto-cerebellar and olivo-cerebellar pathways on motor and spatial learning in the rat.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Fetal and neonatal gene therapy: benefits and pitfalls

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

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.6300249

7 January 2021

Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

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.6300249

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.6300249

7 January 2021

The effect of prenatal hypoxia on brain development: short- and long-term consequences demonstrated in rodent models

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Long-term transgene expression from plasmid DNA gene therapy vectors is negatively affected by CpG dinucleotides

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Scaleable chromatographic purification process for recombinant adeno-associated virus (rAAV).

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

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.6300249

7 January 2021

An improved cupric-silver method for impregnation of axonal and terminal degeneration.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Application of silver degeneration stains for neurotoxicity testing

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300249

7 January 2021

Identifiers

DOI

10.1038/SJ.MT.6300249

1 reference

29 November 2017

1 reference