(Q46455025)

22 December 2017

title

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

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22 December 2017

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11

Edward H Schuchman

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22 December 2017

author name string

Marco A Passini

1

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22 December 2017

Shannon L Macauley

2

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22 December 2017

Michael R Huff

3

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22 December 2017

Tatyana V Taksir

4

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22 December 2017

Jie Bu

5

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22 December 2017

I-Huan Wu

6

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22 December 2017

Peter A Piepenhagen

7

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22 December 2017

James C Dodge

8

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22 December 2017

Lamya S Shihabuddin

9

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22 December 2017

Catherine R O'Riordan

10

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22 December 2017

Gregory R Stewart

12

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22 December 2017

publication date

1 May 2005

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22 December 2017

published in

Molecular Therapy

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22 December 2017

volume

11

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22 December 2017

issue

5

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22 December 2017

page(s)

754-762

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22 December 2017

Acid sphingomyelinase-deficient macrophages have defective cholesterol trafficking and efflux

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7 January 2021

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7 January 2021

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7 January 2021

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Very low levels of high density lipoprotein cholesterol in four sibs of a family with non-neuropathic Niemann-Pick disease and sea-blue histiocytosis

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7 January 2021

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7 January 2021

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Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for canavan disease

7 January 2021

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Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.

7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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7 January 2021

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10.1016/J.YMTHE.2005.01.011

7 January 2021

Identifiers

DOI

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22 December 2017

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22 December 2017