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Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.
Mol Ther. 2016 Feb;24(2):206-216. doi: 10.1038/mt.2015.189. Epub 2015 Oct 8.
Mol Ther. 2016.
PMID: 26447927
Free PMC article.
Analysis of mucolipidosis II/III GNPTAB missense mutations identifies domains of UDP-GlcNAc:lysosomal enzyme GlcNAc-1-phosphotransferase involved in catalytic function and lysosomal enzyme recognition.
Qian Y, van Meel E, Flanagan-Steet H, Yox A, Steet R, Kornfeld S.
Qian Y, et al.
J Biol Chem. 2015 Jan 30;290(5):3045-56. doi: 10.1074/jbc.M114.612507. Epub 2014 Dec 11.
J Biol Chem. 2015.
PMID: 25505245
Free PMC article.
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