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Preclinical and clinical evaluation of the LRRK2 inhibitor DNL201 for Parkinson's disease.
Jennings D, Huntwork-Rodriguez S, Henry AG, Sasaki JC, Meisner R, Diaz D, Solanoy H, Wang X, Negrou E, Bondar VV, Ghosh R, Maloney MT, Propson NE, Zhu Y, Maciuca RD, Harris L, Kay A, LeWitt P, King TA, Kern D, Ellenbogen A, Goodman I, Siderowf A, Aldred J, Omidvar O, Masoud ST, Davis SS, Arguello A, Estrada AA, de Vicente J, Sweeney ZK, Astarita G, Borin MT, Wong BK, Wong H, Nguyen H, Scearce-Levie K, Ho C, Troyer MD. Jennings D, et al. Among authors: nguyen h. Sci Transl Med. 2022 Jun 8;14(648):eabj2658. doi: 10.1126/scitranslmed.abj2658. Epub 2022 Jun 8. Sci Transl Med. 2022. PMID: 35675433
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: nguyen h. 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: nguyen hn. Int J Mol Sci. 2020 Jul 22;21(15):5188. doi: 10.3390/ijms21155188. Int J Mol Sci. 2020. PMID: 32707880 Free PMC article.
Understanding LRRK2 kinase activity in preclinical models and human subjects through quantitative analysis of LRRK2 and pT73 Rab10.
Wang X, Negrou E, Maloney MT, Bondar VV, Andrews SV, Montalban M, Llapashtica C, Maciuca R, Nguyen H, Solanoy H, Arguello A, Przybyla L, Moerke NJ, Huntwork-Rodriguez S, Henry AG. Wang X, et al. Among authors: nguyen h. Sci Rep. 2021 Jun 18;11(1):12900. doi: 10.1038/s41598-021-91943-4. Sci Rep. 2021. PMID: 34145320 Free PMC article.
Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
Logan T, Simon MJ, Rana A, Cherf GM, Srivastava A, Davis SS, Low RLY, Chiu CL, Fang M, Huang F, Bhalla A, Llapashtica C, Prorok R, Pizzo ME, Calvert MEK, Sun EW, Hsiao-Nakamoto J, Rajendra Y, Lexa KW, Srivastava DB, van Lengerich B, Wang J, Robles-Colmenares Y, Kim DJ, Duque J, Lenser M, Earr TK, Nguyen H, Chau R, Tsogtbaatar B, Ravi R, Skuja LL, Solanoy H, Rosen HJ, Boeve BF, Boxer AL, Heuer HW, Dennis MS, Kariolis MS, Monroe KM, Przybyla L, Sanchez PE, Meisner R, Diaz D, Henne KR, Watts RJ, Henry AG, Gunasekaran K, Astarita G, Suh JH, Lewcock JW, DeVos SL, Di Paolo G. Logan T, et al. Among authors: nguyen h. Cell. 2021 Sep 2;184(18):4651-4668.e25. doi: 10.1016/j.cell.2021.08.002. Epub 2021 Aug 26. Cell. 2021. PMID: 34450028 Free PMC article.
Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome.
Arguello A, Meisner R, Thomsen ER, Nguyen HN, Ravi R, Simms J, Lo I, Speckart J, Holtzman J, Gill TM, Chan D, Cheng Y, Chiu CL, Dugas JC, Fang M, Lopez IA, Solanoy H, Tsogtbaatar B, Zhu Y, Bhalla A, Henne KR, Henry AG, Delucchi A, Costanzo S, Harris JM, Diaz D, Scearce-Levie K, Sanchez PE. Arguello A, et al. Among authors: nguyen hn. JCI Insight. 2021 Oct 8;6(19):e145445. doi: 10.1172/jci.insight.145445. JCI Insight. 2021. PMID: 34622797 Free PMC article.
Molecular architecture determines brain delivery of a transferrin receptor-targeted lysosomal enzyme.
Arguello A, Mahon CS, Calvert MEK, Chan D, Dugas JC, Pizzo ME, Thomsen ER, Chau R, Damo LA, Duque J, Fang M, Giese T, Kim DJ, Liang N, Nguyen HN, Solanoy H, Tsogtbaatar B, Ullman JC, Wang J, Dennis MS, Diaz D, Gunasekaran K, Henne KR, Lewcock JW, Sanchez PE, Troyer MD, Harris JM, Scearce-Levie K, Shan L, Watts RJ, Thorne RG, Henry AG, Kariolis MS. Arguello A, et al. Among authors: nguyen hn. J Exp Med. 2022 Mar 7;219(3):e20211057. doi: 10.1084/jem.20211057. Epub 2022 Feb 28. J Exp Med. 2022. PMID: 35226042 Free PMC article.
Novel App knock-in mouse model shows key features of amyloid pathology and reveals profound metabolic dysregulation of microglia.
Xia D, Lianoglou S, Sandmann T, Calvert M, Suh JH, Thomsen E, Dugas J, Pizzo ME, DeVos SL, Earr TK, Lin CC, Davis S, Ha C, Leung AW, Nguyen H, Chau R, Yulyaningsih E, Lopez I, Solanoy H, Masoud ST, Liang CC, Lin K, Astarita G, Khoury N, Zuchero JY, Thorne RG, Shen K, Miller S, Palop JJ, Garceau D, Sasner M, Whitesell JD, Harris JA, Hummel S, Gnörich J, Wind K, Kunze L, Zatcepin A, Brendel M, Willem M, Haass C, Barnett D, Zimmer TS, Orr AG, Scearce-Levie K, Lewcock JW, Di Paolo G, Sanchez PE. Xia D, et al. Among authors: nguyen h. Mol Neurodegener. 2022 Jun 11;17(1):41. doi: 10.1186/s13024-022-00547-7. Mol Neurodegener. 2022. PMID: 35690868 Free PMC article.
Discovery of Potent and Selective Dual Leucine Zipper Kinase/Leucine Zipper-Bearing Kinase Inhibitors with Neuroprotective Properties in In Vitro and In Vivo Models of Amyotrophic Lateral Sclerosis.
Craig RA 2nd, Fox BM, Hu C, Lexa KW, Osipov M, Thottumkara AP, Larhammar M, Miyamoto T, Rana A, Kane LA, Yulyaningsih E, Solanoy H, Nguyen H, Chau R, Earr T, Kajiwara Y, Fleck D, Lucas A, Haddick PCG, Takahashi RH, Tong V, Wang J, Canet MJ, Poda SB, Scearce-Levie K, Srivastava A, Sweeney ZK, Xu M, Zhang R, He J, Lei Y, Zhuo Z, de Vicente J. Craig RA 2nd, et al. Among authors: nguyen h. J Med Chem. 2022 Dec 22;65(24):16290-16312. doi: 10.1021/acs.jmedchem.2c01056. Epub 2022 Dec 5. J Med Chem. 2022. PMID: 36469401
A TREM2-activating antibody with a blood-brain barrier transport vehicle enhances microglial metabolism in Alzheimer's disease models.
van Lengerich B, Zhan L, Xia D, Chan D, Joy D, Park JI, Tatarakis D, Calvert M, Hummel S, Lianoglou S, Pizzo ME, Prorok R, Thomsen E, Bartos LM, Beumers P, Capell A, Davis SS, de Weerd L, Dugas JC, Duque J, Earr T, Gadkar K, Giese T, Gill A, Gnörich J, Ha C, Kannuswamy M, Kim DJ, Kunte ST, Kunze LH, Lac D, Lechtenberg K, Leung AW, Liang CC, Lopez I, McQuade P, Modi A, Torres VO, Nguyen HN, Pesämaa I, Propson N, Reich M, Robles-Colmenares Y, Schlepckow K, Slemann L, Solanoy H, Suh JH, Thorne RG, Vieira C, Wind-Mark K, Xiong K, Zuchero YJY, Diaz D, Dennis MS, Huang F, Scearce-Levie K, Watts RJ, Haass C, Lewcock JW, Di Paolo G, Brendel M, Sanchez PE, Monroe KM. van Lengerich B, et al. Among authors: nguyen hn. Nat Neurosci. 2023 Mar;26(3):416-429. doi: 10.1038/s41593-022-01240-0. Epub 2023 Jan 12. Nat Neurosci. 2023. PMID: 36635496 Free PMC article.
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