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Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients.
Int J Mol Sci. 2020 Jul 22;21(15):5188. doi: 10.3390/ijms21155188.
Int J Mol Sci. 2020.
PMID: 32707880
Free PMC article.
Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice.
Ullman JC, Arguello A, Getz JA, Bhalla A, Mahon CS, Wang J, Giese T, Bedard C, Kim DJ, Blumenfeld JR, Liang N, Ravi R, Nugent AA, Davis SS, Ha C, Duque J, Tran HL, Wells RC, Lianoglou S, Daryani VM, Kwan W, Solanoy H, Nguyen H, Earr T, Dugas JC, Tuck MD, Harvey JL, Reyzer ML, Caprioli RM, Hall S, Poda S, Sanchez PE, Dennis MS, Gunasekaran K, Srivastava A, Sandmann T, Henne KR, Thorne RG, Di Paolo G, Astarita G, Diaz D, Silverman AP, Watts RJ, Sweeney ZK, Kariolis MS, Henry AG.
Ullman JC, et al. Among authors: blumenfeld jr.
Sci Transl Med. 2020 May 27;12(545):eaay1163. doi: 10.1126/scitranslmed.aay1163.
Sci Transl Med. 2020.
PMID: 32461331
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