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Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.
Lagresle-Peyrou C, Lefrère F, Magrin E, Ribeil JA, Romano O, Weber L, Magnani A, Sadek H, Plantier C, Gabrion A, Ternaux B, Félix T, Couzin C, Stanislas A, Tréluyer JM, Lamhaut L, Joseph L, Delville M, Miccio A, André-Schmutz I, Cavazzana M. Lagresle-Peyrou C, et al. Among authors: romano o. Haematologica. 2018 May;103(5):778-786. doi: 10.3324/haematol.2017.184788. Epub 2018 Feb 22. Haematologica. 2018. PMID: 29472357 Free PMC article. Clinical Trial.
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.
Antoniani C, Meneghini V, Lattanzi A, Felix T, Romano O, Magrin E, Weber L, Pavani G, El Hoss S, Kurita R, Nakamura Y, Cradick TJ, Lundberg AS, Porteus M, Amendola M, El Nemer W, Cavazzana M, Mavilio F, Miccio A. Antoniani C, et al. Among authors: romano o. Blood. 2018 Apr 26;131(17):1960-1973. doi: 10.1182/blood-2017-10-811505. Epub 2018 Mar 8. Blood. 2018. PMID: 29519807 Free article.
GATA Factor-Mediated Gene Regulation in Human Erythropoiesis.
Romano O, Petiti L, Felix T, Meneghini V, Portafax M, Antoniani C, Amendola M, Bicciato S, Peano C, Miccio A. Romano O, et al. iScience. 2020 Apr 24;23(4):101018. doi: 10.1016/j.isci.2020.101018. Epub 2020 Mar 30. iScience. 2020. PMID: 32283524 Free PMC article.
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression.
Antoniou P, Hardouin G, Martinucci P, Frati G, Felix T, Chalumeau A, Fontana L, Martin J, Masson C, Brusson M, Maule G, Rosello M, Giovannangeli C, Abramowski V, de Villartay JP, Concordet JP, Del Bene F, El Nemer W, Amendola M, Cavazzana M, Cereseto A, Romano O, Miccio A. Antoniou P, et al. Among authors: romano o. Nat Commun. 2022 Nov 4;13(1):6618. doi: 10.1038/s41467-022-34493-1. Nat Commun. 2022. PMID: 36333351 Free PMC article.
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies.
Brusson M, Chalumeau A, Martinucci P, Romano O, Felix T, Poletti V, Scaramuzza S, Ramadier S, Masson C, Ferrari G, Mavilio F, Cavazzana M, Amendola M, Miccio A. Brusson M, et al. Among authors: romano o. Mol Ther Nucleic Acids. 2023 Mar 22;32:229-246. doi: 10.1016/j.omtn.2023.03.012. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37090420 Free PMC article.
75 results