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Page 1
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: harris jm. JCI Insight. 2021 Oct 8;6(19):e145445. doi: 10.1172/jci.insight.145445. JCI Insight. 2021. PMID: 34622797 Free PMC article.
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: harris jm. Int J Mol Sci. 2020 Jul 22;21(15):5188. doi: 10.3390/ijms21155188. Int J Mol Sci. 2020. PMID: 32707880 Free PMC article.
High-Throughput Liquid Chromatography-Tandem Mass Spectrometry Quantification of Glycosaminoglycans as Biomarkers of Mucopolysaccharidosis II.
Wang J, Bhalla A, Ullman JC, Fang M, Ravi R, Arguello A, Thomsen E, Tsogtbaatar B, Guo JL, Skuja LL, Dugas JC, Davis SS, Poda SB, Gunasekaran K, Costanzo S, Sweeney ZK, Henry AG, Harris JM, Henne KR, Astarita G. Wang J, et al. Among authors: harris jm. Int J Mol Sci. 2020 Jul 30;21(15):5449. doi: 10.3390/ijms21155449. Int J Mol Sci. 2020. PMID: 32751752 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: harris jm. 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.
Longitudinal biomarkers in amyotrophic lateral sclerosis.
Huang F, Zhu Y, Hsiao-Nakamoto J, Tang X, Dugas JC, Moscovitch-Lopatin M, Glass JD, Brown RH Jr, Ladha SS, Lacomis D, Harris JM, Scearce-Levie K, Ho C, Bowser R, Berry JD. Huang F, et al. Among authors: harris jm. Ann Clin Transl Neurol. 2020 Jul;7(7):1103-1116. doi: 10.1002/acn3.51078. Epub 2020 Jun 9. Ann Clin Transl Neurol. 2020. PMID: 32515902 Free PMC article.
Safety, pharmacokinetics and target engagement of novel RIPK1 inhibitor SAR443060 (DNL747) for neurodegenerative disorders: Randomized, placebo-controlled, double-blind phase I/Ib studies in healthy subjects and patients.
Vissers MFJM, Heuberger JAAC, Groeneveld GJ, Oude Nijhuis J, De Deyn PP, Hadi S, Harris J, Tsai RM, Cruz-Herranz A, Huang F, Tong V, Erickson R, Zhu Y, Scearce-Levie K, Hsiao-Nakamoto J, Tang X, Chang M, Fox BM, Estrada AA, Pomponio RJ, Alonso-Alonso M, Zilberstein M, Atassi N, Troyer MD, Ho C. Vissers MFJM, et al. Clin Transl Sci. 2022 Aug;15(8):2010-2023. doi: 10.1111/cts.13317. Epub 2022 Jun 1. Clin Transl Sci. 2022. PMID: 35649245 Free PMC article. Clinical Trial.
Disconnect between sputum neutrophils and other measures of airway inflammation in asthma.
Arron JR, Choy DF, Laviolette M, Kelsen SG, Hatab A, Leigh R, Thomson NC, Bleecker ER, Olivenstein R, Avila PC, Jarjour NN, Castro M, Gauvreau GM, Good JT, Kline JN, Mansur A, Mayers I, Heaney LG, Hamid Q, Harris JM. Arron JR, et al. Among authors: harris jm. Eur Respir J. 2014 Feb;43(2):627-9. doi: 10.1183/09031936.00117013. Epub 2013 Oct 31. Eur Respir J. 2014. PMID: 24176993 Free article. No abstract available.
Reply to Hunsberger and Memoli, "Efficacy Analysis in Healthy-Volunteer Influenza Challenge Trials: Intention To Treat".
McBride JM, Lim JJ, Burgess T, Deng R, Derby MA, Maia M, Horn P, Siddiqui O, Sheinson D, Chen-Harris H, Newton EM, Fillos D, Nazzal D, Rosenberger CM, Ohlson MB, Lambkin-Williams R, Fathi H, Harris JM, Tavel JA. McBride JM, et al. Among authors: harris jm. Antimicrob Agents Chemother. 2017 Dec 21;62(1):e02034-17. doi: 10.1128/AAC.02034-17. Print 2018 Jan. Antimicrob Agents Chemother. 2017. PMID: 29269431 Free PMC article. No abstract available.
746 results