Widespread distribution of β-hexosaminidase activity in the brain of a Sandhoff mouse model after coinjection of adenoviral vector and mannitol-Reference-Cited by-同舟云学术

Widespread distribution of β-hexosaminidase activity in the brain of a Sandhoff mouse model after coinjection of adenoviral vector and mannitol

Published:2003-09-08 Issue:21 Volume:10 Page:1841-1849
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Bourgoin C,Emiliani C,Kremer E J,Gelot A,Tancini B,Gravel R A,Drugan C,Orlacchio A,Poenaru L,Caillaud C

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.nature.com/articles/3302081.pdf

Reference34 articles.

1. Gravel RA et al. The GM2 gangliosidoses. In: Scriver CR, Beaudet AL, Valle D, Sly WS (eds). The Metabolic and Molecular Bases of Inherited Disease. McGraw Hill: New York, 2001, pp. 3827–3876.

2. Phaneuf D et al. Dramatically different phenotypes in mouse models of human Tay–Sachs and Sandhoff diseases. Hum Mol Genet 1996; 5: 1–14.

3. Sango K et al. Mouse models of Tay–Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism. Nat Genet 1995; 11: 170–176.

4. Akli S et al. Transfer of a foreign gene into the brain using adenovirus vectors. Nat Genet 1993; 3: 224–228.

5. Caillaud C et al. Adenoviral vector as a gene delivery system into cultured rat neuronal and glial cells. Eur J Neurosci 1993; 5: 1287–1291.

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