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Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice.
Sci Transl Med. 2020 May 27;12(545):eaay1163. doi: 10.1126/scitranslmed.aay1163.
Sci Transl Med. 2020.
PMID: 32461331
Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients.
Bhalla A, Ravi R, Fang M, Arguello A, Davis SS, Chiu CL, Blumenfeld JR, Nguyen HN, Earr TK, Wang J, Astarita G, Zhu Y, Fiore D, Scearce-Levie K, Diaz D, Cahan H, Troyer MD, Harris JM, Escolar ML.
Bhalla A, et al. Among authors: blumenfeld jr.
Int J Mol Sci. 2020 Jul 22;21(15):5188. doi: 10.3390/ijms21155188.
Int J Mol Sci. 2020.
PMID: 32707880
Free PMC article.
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