Enzyme replacement for GM1-gangliosidosis: Uptake, lysosomal activation, and cellular disease correction using a novel β-galactosidase:RTB lectin fusion-Reference-Cited by-同舟云学术

Enzyme replacement for GM1-gangliosidosis: Uptake, lysosomal activation, and cellular disease correction using a novel β-galactosidase:RTB lectin fusion

Published:2016-02 Issue:2 Volume:117 Page:199-209
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Condori Jose,Acosta Walter,Ayala Jorge,Katta Varun,Flory Ashley,Martin Reid,Radin Jonathan,Cramer Carole L.,Radin David N.

Funder

NIH/NINDS

National Tay-Sachs and Allied Diseases Association

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference70 articles.

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2. Chemokine-induced recruitment of genetically modified bone marrow cells into the CNS of GM1-gangliosidosis mice corrects neuronal pathology;Sano;Blood,2005

3. Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis;Elliot-Smith;Mol. Genet. Metab.,2008

4. Substrate reduction reduces gangliosides in postnatal cerebrum-brainstem and cerebellum in GM1 gangliosidosis mice;Kasperzyk;J. Lipid Res.,2005

5. N-butyldeoxygalactonojirimycin reduces neonatal brain ganglioside content in a mouse model of GM1 gangliosidosis;Kasperzyk;J. Neurochem.,2004

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