Acid ceramidase deficiency: Farber disease and SMA-PME von Yu, Fabian P S, Amintas, Samuel, Levade, Thierry, Medin, Jeffrey A

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Lentivirus-mediated gene therapy for Fabry disease von Khan, Aneal, Barber, Dwayne L., Huang, Ju, Rupar, C. Anthony, Rip, Jack W., Auray-Blais, Christiane, Boutin, Michel, O’Hoski, Pamela, Gargulak, Kristy, McKillop, William M., Fraser, Graeme, Wasim, Syed, LeMoine, Kaye, Jelinski, Shelly, Chaudhry, Ahsan, Prokopishyn, Nicole, Morel, Chantal F., Couban, Stephen, Duggan, Peter R., Fowler, Daniel H., Keating, Armand, West, Michael L., Foley, Ronan, Medin, Jeffrey A.

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

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The engineered thymidylate kinase (TMPK)/AZT enzyme-prodrug axis offers efficient bystander cell killing for suicide gene therapy of cancer von Sato, Takeya, Neschadim, Anton, Lavie, Arnon, Yanagisawa, Teruyuki, Medin, Jeffrey A

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On-Tissue Localization of Ceramides and Other Sphingolipids by MALDI Mass Spectrometry Imaging von Jones, E. Ellen, Dworski, Shaalee, Canals, Daniel, Casas, Josefina, Fabrias, Gemma, Schoenling, Drew, Levade, Thierry, Denlinger, Chadrick, Hannun, Yusuf A, Medin, Jeffrey A, Drake, Richard R

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Sarcoma IL-12 overexpression facilitates NK cell immunomodulation von Rademacher, Mary Jo, Cruz, Anahi, Faber, Mary, Oldham, Robyn A. A., Wang, Dandan, Medin, Jeffrey A., Schloemer, Nathan J.

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Ceramide metabolism alterations contribute to Tumor Necrosis Factor-induced melanoma dedifferentiation and predict resistance to immune checkpoint inhibitors in advanced melanoma p... von Dufau, Carine, Genais, Matthieu, Mucher, Elodie, Jung, Benjamin, Garcia, Virginie, Montfort, Anne, Tosolini, Marie, Clarke, Christopher J, Medin, Jeffrey A, Levade, Thierry, Delord, Jean-Pierre, Meyer, Nicolas, Pancaldi, Vera, Andrieu-Abadie, Nathalie, Ségui, Bruno

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Neuraminidase 1 activates insulin receptor and reverses insulin resistance in obese mice von Fougerat, Anne, Pan, Xuefang, Smutova, Victoria, Heveker, Nikolaus, Cairo, Christopher W., Issad, Tarik, Larrivée, Bruno, Medin, Jeffrey A., Pshezhetsky, Alexey V.

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Acid Ceramidase Deficiency: Bridging Gaps between Clinical Presentation, Mouse Models, and Future Therapeutic Interventions von Kleynerman, Annie, Rybova, Jitka, Faber, Mary L, McKillop, William M, Levade, Thierry, Medin, Jeffrey A

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Autologous, lentivirus‐modified, T‐rapa cell “micropharmacies” for lysosomal storage disorders von Nagree, Murtaza S, Felizardo, Tania C, Faber, Mary L, Rybova, Jitka, Rupar, C Anthony, Foley, S Ronan, Fuller, Maria, Fowler, Daniel H, Medin, Jeffrey A

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Novel anti-CD30/CD3 bispecific antibodies activate human T cells and mediate potent anti-tumor activity von Faber, Mary L, Oldham, Robyn A A, Thakur, Archana, Rademacher, Mary Jo, Kubicka, Ewa, Dlugi, Theresa A, Gifford, Steven A, McKillop, William M, Schloemer, Nathan J, Lum, Lawrence G, Medin, Jeffrey A

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Systemic ceramide accumulation leads to severe and varied pathological consequences von Alayoubi, Abdulfatah M., Wang, James C. M., Au, Bryan C. Y., Carpentier, Stéphane, Garcia, Virginie, Dworski, Shaalee, El‐Ghamrasni, Samah, Kirouac, Kevin N., Exertier, Mathilde J., Xiong, Zi Jian, Privé, Gilbert G., Simonaro, Calogera M., Casas, Josefina, Fabrias, Gemma, Schuchman, Edward H., Turner, Patricia V., Hakem, Razqallah, Levade, Thierry, Medin, Jeffrey A.

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Lentivirus‐mediated gene therapy for Fabry disease: 5‐year End‐of‐Study results from the Canadian FACTs trial von Khan, Aneal, Barber, Dwayne L., McKillop, William M., Rupar, C. Anthony, Auray‐Blais, Christiane, Fraser, Graeme, Fowler, Daniel H., Berger, Alexandra, Foley, Ronan, Keating, Armand, West, Michael L., Medin, Jeffrey A.

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Natural killer cell lines preferentially kill clonogenic multiple myeloma cells and decrease myeloma engraftment in a bioluminescent xenograft mouse model von SWIFT, Brenna E, WILLIAMS, Brent A, KOSAKA, Yoko, WANG, Xing-Hua, MEDIN, Jeffrey A, VISWANATHAN, Sowmya, MARTINEZ-LOPEZ, Joaquin, KEATING, Armand

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Adiponectin overexpression improves metabolic abnormalities caused by acid ceramidase deficiency but does not prolong lifespan in a mouse model of Farber Disease von Norris, Marie K., Tippetts, Trevor S., Wilkerson, Joseph L., Nicholson, Rebekah J., Maschek, J. Alan, Levade, Thierry, Medin, Jeffrey A., Summers, Scott A., Holland, William L.

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Azidothymidine-triphosphate impairs mitochondrial dynamics by disrupting the quality control system von Nomura, Ryosuke, Sato, Takeya, Sato, Yuka, Medin, Jeffrey A., Kushimoto, Shigeki, Yanagisawa, Teruyuki

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Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease von Saleh, Amr H., Rothe, Michael, Barber, Dwayne L., McKillop, William M., Fraser, Graeme, Morel, Chantal F., Schambach, Axel, Auray-Blais, Christiane, West, Michael L., Khan, Aneal, Fowler, Daniel H., Rupar, C. Anthony, Foley, Ronan, Medin, Jeffrey A., Keating, Armand

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Spinal muscular atrophy-like phenotype in a mouse model of acid ceramidase deficiency von Nagree, Murtaza S., Rybova, Jitka, Kleynerman, Annie, Ahrenhoerster, Carissa J., Saville, Jennifer T., Xu, TianMeng, Bachochin, Maxwell, McKillop, William M., Lawlor, Michael W., Pshezhetsky, Alexey V., Isaeva, Olena, Budde, Matthew D., Fuller, Maria, Medin, Jeffrey A.

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Discovery and validation of surface N-glycoproteins in MM cell lines and patient samples uncovers immunotherapy targets von Oldham, Robyn A A, Faber, Mary L, Keppel, Theodore R, Buchberger, Amanda R, Waas, Matthew, Hari, Parameswaran, Gundry, Rebekah L, Medin, Jeffrey A

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Yin and Yang of mesenchymal stem cells and aplastic anemia von Broglie, Larisa, Margolis, David, Medin, Jeffrey A

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