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Molecular Therapy - Methods and Clinical Development, ‎2329-0501

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  1. Induced dendritic cells co-expressing GM-CSF/IFN-α/tWT1 priming T and B cells and automated manufacturing to boost GvL

    Bialek-Waldmann, J. K., Domning, S., Esser, R., Glienke, W., Mertens, M., Aleksandrova, K., Arseniev, L., Kumar, S., Schneider, A., Koenig, J., Theobald, S. J., Tsay, H. C., Cornelius, A. D. A., Bonifacius, A., Eiz-Vesper, B., Figueiredo, C., Schaudien, D., Talbot, S. R., Bleich, A., Spineli, L. M. & 8 others, von Kaisenberg, C., Clark, C., Blasczyk, R., Heuser, M., Ganser, A., Köhl, U., Farzaneh, F. & Stripecke, R., 11 Jun 2021, In: Molecular Therapy - Methods and Clinical Development. 21, p. 621-641 21 p.

    Research output: Contribution to journalArticlepeer-review

  2. Deferoxamine mesylate improves splicing and GAA activity of the common c.-32-13T>G allele in late-onset PD patient fibroblasts

    Buratti, E., Peruzzo, P., Braga, L., Zanin, I., Stuani, C., Goina, E., Romano, M., Giacca, M. & Dardis, A., 12 Mar 2021, In: Molecular Therapy - Methods and Clinical Development. 20, p. 227-236 10 p.

    Research output: Contribution to journalArticlepeer-review

  3. Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

    Ellison, S. M., Liao, A., Wood, S., Taylor, J., Youshani, A. S., Rowlston, S., Parker, H., Armant, M., Biffi, A., Chan, L., Farzaneh, F., Wynn, R., Jones, S. A., Heal, P., Gaspar, H. B. & Bigger, B. W., 14 Jun 2019, In: Molecular Therapy - Methods and Clinical Development. 13, p. 399-413 15 p.

    Research output: Contribution to journalArticlepeer-review

  4. Lentiviral vector purification using genetically encoded biotin mimic in packaging cell

    Mekkaoui, L., Parekh, F., Kotsopoulou, E., Darling, D., Dickson, G., Cheung, G. W., Chan, L., MacLellan-Gibson, K., Mattiuzzo, G., Farzaneh, F., Takeuchi, Y. & Pule, M., 23 Oct 2018, (E-pub ahead of print) In: Molecular Therapy - Methods and Clinical Development.

    Research output: Contribution to journalArticlepeer-review

  5. A Rapamycin-Based GMP-Compatible Process for the Isolation and Expansion of Regulatory T Cells for Clinical Trials

    Fraser, H., Safinia, N., Grageda, N., Thirkell, S., Lowe, K., Fry, L. J., Scottá, C., Hope, A., Fisher, C., Hilton, R., Game, D., Harden, P., Bushell, A., Wood, K., Lechler, R. I. & Lombardi, G., 16 Mar 2018, In: Molecular Therapy - Methods and Clinical Development. 8, p. 198-209 12 p.

    Research output: Contribution to journalArticlepeer-review

  6. An optimized lentiviral vector corrects efficiently the human sickle cell disease phenotype

    Weber, L., Poletti, V., Magrin, E., Antoniani, C., Martin, S., Bayard, C., Sadek, H., Felix, T., Meneghini, V., Antoniou, M. N., El- Nemer, W., Mavilio, F., Cavazzana, M., Andre-Schmutz, I. & Miccio, A., 2018, In: Molecular Therapy - Methods and Clinical Development.

    Research output: Contribution to journalArticlepeer-review

  7. A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I

    Mendez, D. C., Stover, A. E., Rangel, A. D., Brick, D. J., Nethercott, H. E., Torres, M. A., Khalid, O., Wong, A. MS., Cooper, J. D., Jester, J. V., Monuki, E. S., McGuire, C., Le, S. Q., Kan, S. H., Dickson, P. I. & Schwartz, P. H., 14 Dec 2016, (E-pub ahead of print) In: Molecular Therapy - Methods and Clinical Development. 2, p. 14068 1 p.

    Research output: Contribution to journalArticlepeer-review

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