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Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA.
Ellison SM, Liao A, Wood S, Taylor J, Youshani AS, Rowlston S, Parker H, Armant M, Biffi A, Chan L, Farzaneh F, Wynn R, Jones SA, Heal P, Gaspar HB, Bigger BW. Ellison SM, et al. Mol Ther Methods Clin Dev. 2019 Apr 6;13:399-413. doi: 10.1016/j.omtm.2019.04.001. eCollection 2019 Jun 14. Mol Ther Methods Clin Dev. 2019. PMID: 31044143 Free PMC article.
Vesicular stomatitis virus glycoprotein- and Venezuelan equine encephalitis virus-derived glycoprotein-pseudotyped lentivirus vectors differentially transduce corneal endothelium, trabecular meshwork, and human photoreceptors.
Lipinski DM, Barnard AR, Charbel Issa P, Singh MS, De Silva SR, Trabalza A, Eleftheriadou I, Ellison SM, Mazarakis ND, MacLaren RE. Lipinski DM, et al. Among authors: ellison sm. Hum Gene Ther. 2014 Jan;25(1):50-62. doi: 10.1089/hum.2013.009. Epub 2013 Dec 21. Hum Gene Ther. 2014. PMID: 24125177
261 results