Towards in vivo amplification: Overcoming hurdles in the use of hematopoietic stem cells in transplantation and gene therapy

Bằng Nagree, Murtaza S, López-Vásquez, Lucía, Medin, Jeffrey A
Xuất bản năm World J Stem Cells (2015)

Acid Ceramidase Deficiency in Mice Leads to Severe Ocular Pathology and Visual Impairment

Bằng Yu, Fabian P.S., Sajdak, Benjamin S., Sikora, Jakub, Salmon, Alexander E., Nagree, Murtaza S., Gurka, Jiří, Kassem, Iris S., Lipinski, Daniel M., Carroll, Joseph, Medin, Jeffrey A.
Xuất bản năm Am J Pathol (2019)

Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target

Bằng Li, Yedda, Xu, Yue, Benitez, Bruno A., Nagree, Murtaza S., Dearborn, Joshua T., Jiang, Xuntian, Guzman, Miguel A., Woloszynek, Josh C., Giaramita, Alex, Yip, Bryan K., Elsbernd, Joseph, Babcock, Michael C., Lo, Melanie, Fowler, Stephen C., Wozniak, David F., Vogler, Carole A., Medin, Jeffrey A., Crawford, Brett E., Sands, Mark S.
Xuất bản năm Proc Natl Acad Sci U S A (2019)

Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34(+) Cells for Correction of Fabry Disease

Bằng Huang, Ju, Khan, Aneal, Au, Bryan C., Barber, Dwayne L., López-Vásquez, Lucía, Prokopishyn, Nicole L., Boutin, Michel, Rothe, Michael, Rip, Jack W., Abaoui, Mona, Nagree, Murtaza S., Dworski, Shaalee, Schambach, Axel, Keating, Armand, West, Michael L., Klassen, John, Turner, Patricia V., Sirrs, Sandra, Rupar, C. Anthony, Auray-Blais, Christiane, Foley, Ronan, Medin, Jeffrey A.
Xuất bản năm Mol Ther Methods Clin Dev (2017)