AAV-Mediated Gene Delivery in Adult GM1- Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival.
- Published in:
- PLoS ONE, 2010, v. 5, n. 10, p. 1, doi. 10.1371/journal.pone.0013468
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- Publication type:
- Article
Works matching DE "TREATMENT of Tay-Sachs disease"
Results: 3
1
2
CT and MRI findings in a case of infantile form of GM2 gangliosidosis: Tay-Sachs disease.
- Published in:
- 2016
- By:
- Publication type:
- journal article
3
A Bicyclic 1-Deoxygalactonojirimycin Derivative as a Novel Pharmacological Chaperone for GM<sub>1</sub> Gangliosidosis.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 3, p. 526, doi. 10.1038/mt.2012.263
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- Publication type:
- Article