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Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype.
Weber L, Frati G, Felix T, Hardouin G, Casini A, Wollenschlaeger C, Meneghini V, Masson C, De Cian A, Chalumeau A, Mavilio F, Amendola M, Andre-Schmutz I, Cereseto A, El Nemer W, Concordet JP, Giovannangeli C, Cavazzana M, Miccio A. Weber L, et al. Among authors: cavazzana m. Sci Adv. 2020 Feb 12;6(7):eaay9392. doi: 10.1126/sciadv.aay9392. Print 2020 Feb. Sci Adv. 2020. PMID: 32917636 Free PMC article.
Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination.
André-Schmutz I, Dal-Cortivo L, Six E, Kaltenbach S, Cocchiarella F, Le Chenadec J, Cagnard N, Cordier AG, Benachi A, Mandelbrot L, Azria E, Bouallag N, Luce S, Ternaux B, Reimann C, Revy P, Radford-Weiss I, Leschi C, Recchia A, Mavilio F, Cavazzana M, Blanche S. André-Schmutz I, et al. Among authors: cavazzana m. J Infect Dis. 2013 Jul 15;208(2):235-43. doi: 10.1093/infdis/jit149. Epub 2013 Apr 4. J Infect Dis. 2013. PMID: 23559464
Gene-corrected human Munc13-4-deficient CD8+ T cells can efficiently restrict EBV-driven lymphoproliferation in immunodeficient mice.
Soheili T, Rivière J, Ricciardelli I, Durand A, Verhoeyen E, Derrien AC, Lagresle-Peyrou C, de Saint Basile G, Cosset FL, Amrolia P, André-Schmutz I, Cavazzana M. Soheili T, et al. Among authors: cavazzana m. Blood. 2016 Dec 15;128(24):2859-2862. doi: 10.1182/blood-2016-07-729871. Epub 2016 Oct 31. Blood. 2016. PMID: 27799161 Free article. No abstract available.
Gene Therapy in a Patient with Sickle Cell Disease.
Ribeil JA, Hacein-Bey-Abina S, Payen E, Magnani A, Semeraro M, Magrin E, Caccavelli L, Neven B, Bourget P, El Nemer W, Bartolucci P, Weber L, Puy H, Meritet JF, Grevent D, Beuzard Y, Chrétien S, Lefebvre T, Ross RW, Negre O, Veres G, Sandler L, Soni S, de Montalembert M, Blanche S, Leboulch P, Cavazzana M. Ribeil JA, et al. Among authors: cavazzana m. N Engl J Med. 2017 Mar 2;376(9):848-855. doi: 10.1056/NEJMoa1609677. N Engl J Med. 2017. PMID: 28249145 Free article.
Gene Therapy for β-Hemoglobinopathies.
Cavazzana M, Antoniani C, Miccio A. Cavazzana M, et al. Mol Ther. 2017 May 3;25(5):1142-1154. doi: 10.1016/j.ymthe.2017.03.024. Epub 2017 Apr 1. Mol Ther. 2017. PMID: 28377044 Free PMC article. Review.
[Gene therapy for sickle cell disease].
Ribeil JA, Blanche S, Cavazzana M. Ribeil JA, et al. Among authors: cavazzana m. Med Sci (Paris). 2017 May;33(5):463-465. doi: 10.1051/medsci/20173305002. Epub 2017 Jun 14. Med Sci (Paris). 2017. PMID: 28612714 Free article. French. No abstract available.
Gene Therapy Approaches to Hemoglobinopathies.
Ferrari G, Cavazzana M, Mavilio F. Ferrari G, et al. Among authors: cavazzana m. Hematol Oncol Clin North Am. 2017 Oct;31(5):835-852. doi: 10.1016/j.hoc.2017.06.010. Hematol Oncol Clin North Am. 2017. PMID: 28895851 Review.
Generation of adult human T-cell progenitors for immunotherapeutic applications.
Simons L, Ma K, de Chappedelaine C, Moiranghtem RD, Elkaim E, Olivré J, Susini S, Appourchaux K, Reimann C, Sadek H, Pellé O, Cagnard N, Magrin E, Lagresle-Peyrou C, Taghon T, Rausell A, Cavazzana M, André-Schmutz I. Simons L, et al. Among authors: cavazzana m. J Allergy Clin Immunol. 2018 Apr;141(4):1491-1494.e4. doi: 10.1016/j.jaci.2017.10.034. Epub 2017 Dec 5. J Allergy Clin Immunol. 2018. PMID: 29208547 Free PMC article. No abstract available.
167 results