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427 results

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Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment.
Navarro S, Quintana-Bustamante O, Sanchez-Dominguez R, Lopez-Manzaneda S, Ojeda-Perez I, Garcia-Torralba A, Alberquilla O, Law K, Beard BC, Bastone A, Rothe M, Villanueva M, Ramirez JC, Fañanas-Baquero S, Nieto-Romero V, Molinos-Vicente A, Gutierrez S, Nicoletti E, García-Bravo M, Bueren JA, Schwartz JD, Segovia JC. Navarro S, et al. Among authors: rothe m. Mol Ther Methods Clin Dev. 2021 Jul 29;22:350-359. doi: 10.1016/j.omtm.2021.07.006. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34514027 Free PMC article.
Preclinical safety and efficacy of an anti-HIV-1 lentiviral vector containing a short hairpin RNA to CCR5 and the C46 fusion inhibitor.
Wolstein O, Boyd M, Millington M, Impey H, Boyer J, Howe A, Delebecque F, Cornetta K, Rothe M, Baum C, Nicolson T, Koldej R, Zhang J, Keech N, Camba Colón J, Breton L, Bartlett J, An DS, Chen IS, Burke B, Symonds GP. Wolstein O, et al. Among authors: rothe m. Mol Ther Methods Clin Dev. 2014 Feb 12;1:11. doi: 10.1038/mtm.2013.11. eCollection 2014. Mol Ther Methods Clin Dev. 2014. PMID: 26015947 Free PMC article.
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID.
Charrier S, Lagresle-Peyrou C, Poletti V, Rothe M, Cédrone G, Gjata B, Mavilio F, Fischer A, Schambach A, de Villartay JP, Cavazzana M, Hacein-Bey-Abina S, Galy A. Charrier S, et al. Among authors: rothe m. Mol Ther Methods Clin Dev. 2019 Sep 13;15:232-245. doi: 10.1016/j.omtm.2019.08.014. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31720302 Free PMC article.
Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy.
Brendel C, Negre O, Rothe M, Guda S, Parsons G, Harris C, McGuinness M, Abriss D, Tsytsykova A, Klatt D, Bentler M, Pellin D, Christiansen L, Schambach A, Manis J, Trebeden-Negre H, Bonner M, Esrick E, Veres G, Armant M, Williams DA. Brendel C, et al. Among authors: rothe m. Mol Ther Methods Clin Dev. 2020 Mar 17;17:589-600. doi: 10.1016/j.omtm.2020.03.015. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32300607 Free PMC article.
Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector.
Dahl M, Smith EMK, Warsi S, Rothe M, Ferraz MJ, Aerts JMFG, Golipour A, Harper C, Pfeifer R, Pizzurro D, Schambach A, Mason C, Karlsson S. Dahl M, et al. Among authors: rothe m. Mol Ther Methods Clin Dev. 2020 Dec 3;20:312-323. doi: 10.1016/j.omtm.2020.11.018. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33511245 Free PMC article.
Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning.
Schwarzer A, Talbot SR, Selich A, Morgan M, Schott JW, Dittrich-Breiholz O, Bastone AL, Weigel B, Ha TC, Dziadek V, Gijsbers R, Thrasher AJ, Staal FJT, Gaspar HB, Modlich U, Schambach A, Rothe M. Schwarzer A, et al. Among authors: rothe m. Mol Ther. 2021 Dec 1;29(12):3383-3397. doi: 10.1016/j.ymthe.2021.06.017. Epub 2021 Jun 24. Mol Ther. 2021. PMID: 34174440 Free PMC article.
Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry Disease.
Huang J, Khan A, Au BC, Barber DL, López-Vásquez L, Prokopishyn NL, Boutin M, Rothe M, Rip JW, Abaoui M, Nagree MS, Dworski S, Schambach A, Keating A, West ML, Klassen J, Turner PV, Sirrs S, Rupar CA, Auray-Blais C, Foley R, Medin JA. Huang J, et al. Among authors: rothe m. Mol Ther Methods Clin Dev. 2017 May 12;5:241-258. doi: 10.1016/j.omtm.2017.05.003. eCollection 2017 Jun 16. Mol Ther Methods Clin Dev. 2017. PMID: 28603745 Free PMC article.
Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.
Brendel C, Rothe M, Santilli G, Charrier S, Stein S, Kunkel H, Abriss D, Müller-Kuller U, Gaspar B, Modlich U, Galy A, Schambach A, Thrasher AJ, Grez M. Brendel C, et al. Among authors: rothe m. Hum Gene Ther Clin Dev. 2018 Jun;29(2):69-79. doi: 10.1089/humc.2017.245. Epub 2018 Apr 17. Hum Gene Ther Clin Dev. 2018. PMID: 29664709
427 results