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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: medin ja. 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.
Lentivirus-mediated gene therapy for Fabry disease.
Khan A, Barber DL, Huang J, Rupar CA, Rip JW, Auray-Blais C, Boutin M, O'Hoski P, Gargulak K, McKillop WM, Fraser G, Wasim S, LeMoine K, Jelinski S, Chaudhry A, Prokopishyn N, Morel CF, Couban S, Duggan PR, Fowler DH, Keating A, West ML, Foley R, Medin JA. Khan A, et al. Among authors: medin ja. Nat Commun. 2021 Feb 25;12(1):1178. doi: 10.1038/s41467-021-21371-5. Nat Commun. 2021. PMID: 33633114 Free PMC article. Clinical Trial.
Preselective gene therapy for Fabry disease.
Qin G, Takenaka T, Telsch K, Kelley L, Howard T, Levade T, Deans R, Howard BH, Malech HL, Brady RO, Medin JA. Qin G, et al. Among authors: medin ja. Proc Natl Acad Sci U S A. 2001 Mar 13;98(6):3428-33. doi: 10.1073/pnas.061020598. Epub 2001 Mar 6. Proc Natl Acad Sci U S A. 2001. PMID: 11248095 Free PMC article.
Gene therapy for Fabry disease.
Siatskas C, Medin JA. Siatskas C, et al. Among authors: medin ja. J Inherit Metab Dis. 2001;24 Suppl 2:25-41; discussion 11-2. doi: 10.1023/a:1012455421014. J Inherit Metab Dis. 2001. PMID: 11758676 Review.
171 results