Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models-Reference-Cited by-同舟云学术

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Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models

Published:2010-03-04 Issue:11 Volume:19 Page:2208-2227
ISSN:1460-2083
Container-title:Human Molecular Genetics
language:en
Short-container-title:

Author:

Lattanzi Annalisa,Neri Margherita,Maderna Claudio,di Girolamo Ilaria,Martino Sabata,Orlacchio Aldo,Amendola Mario,Naldini Luigi,Gritti Angela

Publisher

Oxford University Press (OUP)

Subject

Genetics(clinical),Genetics,Molecular Biology,General Medicine

Link

http://academic.oup.com/hmg/article-pdf/19/11/2208/13905586/ddq099.pdf

Reference90 articles.

1. Lysosomal storage disorders;Vellodi;Br. J. Haematol,2005

2. Globoid cell leucodystrophy (Krabbe's disease): deficiency of galactocerebroside beta-galactosidase;Suzuki;Proc. Natl Acad. Sci. USA,1970

3. Genetics of metachromatic leukodystrophy;Gieselmann;Gene Ther.,1994

4. CNS-directed gene therapy for lysosomal storage diseases;Sands;Acta. Paediatr. Suppl.,2008

5. Complete correction of enzymatic deficiency and neurochemistry in the GM1-gangliosidosis mouse brain by neonatal adeno-associated virus-mediated gene delivery;Broekman;Mol. Ther.,2007

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