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Acid Ceramidase Deficiency in Mice Results in a Broad Range of Central Nervous System Abnormalities.
Sikora J, Dworski S, Jones EE, Kamani MA, Micsenyi MC, Sawada T, Le Faouder P, Bertrand-Michel J, Dupuy A, Dunn CK, Xuan ICY, Casas J, Fabrias G, Hampson DR, Levade T, Drake RR, Medin JA, Walkley SU. Sikora J, et al. Among authors: medin ja. Am J Pathol. 2017 Apr;187(4):864-883. doi: 10.1016/j.ajpath.2016.12.005. Am J Pathol. 2017. PMID: 28342444 Free PMC article.
An update on gene therapy for lysosomal storage disorders.
Nagree MS, Scalia S, McKillop WM, Medin JA. Nagree MS, et al. Among authors: medin ja. 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.
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.
170 results