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The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup.
Brooks PJ, Ottinger EA, Portero D, Lomash RM, Alimardanov A, Terse P, Xu X, Chandler RJ, Geist Hauserman J, Esposito E, Bönnemann CG, Venditti CP, Austin CP, Pariser A, Lo DC. Brooks PJ, et al. Among authors: xu x. Hum Gene Ther. 2020 Oct;31(19-20):1034-1042. doi: 10.1089/hum.2020.259. Hum Gene Ther. 2020. PMID: 32993373 Free PMC article. No abstract available.
Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease.
Ottinger EA, Kao ML, Carrillo-Carrasco N, Yanjanin N, Shankar RK, Janssen M, Brewster M, Scott I, Xu X, Cradock J, Terse P, Dehdashti SJ, Marugan J, Zheng W, Portilla L, Hubbs A, Pavan WJ, Heiss J, Vite CH, Walkley SU, Ory DS, Silber SA, Porter FD, Austin CP, McKew JC. Ottinger EA, et al. Among authors: xu x. Curr Top Med Chem. 2014;14(3):330-9. doi: 10.2174/1568026613666131127160118. Curr Top Med Chem. 2014. PMID: 24283970 Free PMC article. Review.
ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice.
Abera MB, Xiao J, Nofziger J, Titus S, Southall N, Zheng W, Moritz KE, Ferrer M, Cherry JJ, Androphy EJ, Wang A, Xu X, Austin C, Fischbeck KH, Marugan JJ, Burnett BG. Abera MB, et al. Among authors: xu x. JCI Insight. 2016 Nov 17;1(19):e88427. doi: 10.1172/jci.insight.88427. JCI Insight. 2016. PMID: 27882347 Free PMC article.
Safety, pharmacokinetics and sialic acid production after oral administration of N-acetylmannosamine (ManNAc) to subjects with GNE myopathy.
Xu X, Wang AQ, Latham LL, Celeste F, Ciccone C, Malicdan MC, Goldspiel B, Terse P, Cradock J, Yang N, Yorke S, McKew JC, Gahl WA, Huizing M, Carrillo N. Xu X, et al. Mol Genet Metab. 2017 Sep;122(1-2):126-134. doi: 10.1016/j.ymgme.2017.04.010. Epub 2017 Apr 26. Mol Genet Metab. 2017. PMID: 28641925 Free PMC article. Clinical Trial.
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial.
Ory DS, Ottinger EA, Farhat NY, King KA, Jiang X, Weissfeld L, Berry-Kravis E, Davidson CD, Bianconi S, Keener LA, Rao R, Soldatos A, Sidhu R, Walters KA, Xu X, Thurm A, Solomon B, Pavan WJ, Machielse BN, Kao M, Silber SA, McKew JC, Brewer CC, Vite CH, Walkley SU, Austin CP, Porter FD. Ory DS, et al. Among authors: xu x. Lancet. 2017 Oct 14;390(10104):1758-1768. doi: 10.1016/S0140-6736(17)31465-4. Epub 2017 Aug 10. Lancet. 2017. PMID: 28803710 Free PMC article. Clinical Trial.
The Fungal Cyp51 Inhibitor VT-1129 Is Efficacious in an Experimental Model of Cryptococcal Meningitis.
Wiederhold NP, Najvar LK, Garvey EP, Brand SR, Xu X, Ottinger EA, Alimardanov A, Cradock J, Behnke M, Hoekstra WJ, Schotzinger RJ, Jaramillo R, Olivo M, Kirkpatrick WR, Patterson TF. Wiederhold NP, et al. Among authors: xu x. Antimicrob Agents Chemother. 2018 Aug 27;62(9):e01071-18. doi: 10.1128/AAC.01071-18. Print 2018 Sep. Antimicrob Agents Chemother. 2018. PMID: 29987152 Free PMC article.
In Vivo Efficacy of VT-1129 against Experimental Cryptococcal Meningitis with the Use of a Loading Dose-Maintenance Dose Administration Strategy.
Wiederhold NP, Xu X, Wang A, Najvar LK, Garvey EP, Ottinger EA, Alimardanov A, Cradock J, Behnke M, Hoekstra WJ, Brand SR, Schotzinger RJ, Jaramillo R, Olivo M, Kirkpatrick WR, Patterson TF. Wiederhold NP, et al. Among authors: xu x. Antimicrob Agents Chemother. 2018 Oct 24;62(11):e01315-18. doi: 10.1128/AAC.01315-18. Print 2018 Nov. Antimicrob Agents Chemother. 2018. PMID: 30104280 Free PMC article.
Application of niclosamide and analogs as small molecule inhibitors of Zika virus and SARS-CoV-2 infection.
Shamim K, Xu M, Hu X, Lee EM, Lu X, Huang R, Shah P, Xu X, Chen CZ, Shen M, Guo H, Chen L, Itkin Z, Eastman RT, Shinn P, Klumpp-Thomas C, Michael S, Simeonov A, Lo DC, Ming GL, Song H, Tang H, Zheng W, Huang W. Shamim K, et al. Among authors: xu m, xu x. Bioorg Med Chem Lett. 2021 May 15;40:127906. doi: 10.1016/j.bmcl.2021.127906. Epub 2021 Mar 6. Bioorg Med Chem Lett. 2021. PMID: 33689873 Free PMC article.
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