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Is SGSH heterozygosity a risk factor for early-onset neurodegenerative disease?
J Inherit Metab Dis. 2021 May;44(3):763-776. doi: 10.1002/jimd.12359. Epub 2021 Jan 25.
J Inherit Metab Dis. 2021.
PMID: 33423317
AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains.
Hocquemiller M, Hemsley KM, Douglass ML, Tamang SJ, Neumann D, King BM, Beard H, Trim PJ, Winner LK, Lau AA, Snel MF, Gomila C, Ausseil J, Mei X, Giersch L, Plavsic M, Laufer R.
Hocquemiller M, et al. Among authors: douglass ml.
Mol Ther Methods Clin Dev. 2019 Dec 10;17:174-187. doi: 10.1016/j.omtm.2019.12.001. eCollection 2020 Jun 12.
Mol Ther Methods Clin Dev. 2019.
PMID: 31909089
Free PMC article.
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