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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: nagree ms. 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.
An update on gene therapy for lysosomal storage disorders.
Nagree MS, Scalia S, McKillop WM, Medin JA. Nagree MS, et al. Expert Opin Biol Ther. 2019 Jul;19(7):655-670. doi: 10.1080/14712598.2019.1607837. Epub 2019 May 6. Expert Opin Biol Ther. 2019. PMID: 31056978 Review.
Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target.
Li Y, Xu Y, Benitez BA, Nagree MS, Dearborn JT, Jiang X, Guzman MA, Woloszynek JC, Giaramita A, Yip BK, Elsbernd J, Babcock MC, Lo M, Fowler SC, Wozniak DF, Vogler CA, Medin JA, Crawford BE, Sands MS. Li Y, et al. Among authors: nagree ms. Proc Natl Acad Sci U S A. 2019 Oct 1;116(40):20097-20103. doi: 10.1073/pnas.1912108116. Epub 2019 Sep 16. Proc Natl Acad Sci U S A. 2019. PMID: 31527255 Free PMC article.