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Page 1
An Optimized Lentiviral Vector Efficiently Corrects 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 MN, El-Nemer W, Mavilio F, Cavazzana M, Andre-Schmutz I, Miccio A. Weber L, et al. Among authors: mavilio f. Mol Ther Methods Clin Dev. 2018 Aug 4;10:268-280. doi: 10.1016/j.omtm.2018.07.012. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30140714 Free PMC article.
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: mavilio f. Sci Adv. 2020 Feb 12;6(7):eaay9392. doi: 10.1126/sciadv.aay9392. Print 2020 Feb. Sci Adv. 2020. PMID: 32917636 Free PMC article.
Interactions between Retroviruses and the Host Cell Genome.
Poletti V, Mavilio F. Poletti V, et al. Among authors: mavilio f. Mol Ther Methods Clin Dev. 2017 Oct 5;8:31-41. doi: 10.1016/j.omtm.2017.10.001. eCollection 2018 Mar 16. Mol Ther Methods Clin Dev. 2017. PMID: 29159201 Free PMC article. Review.
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease.
Poletti V, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M, Schambach A, Kohn DB, Mavilio F. Poletti V, et al. Among authors: mavilio f. Mol Ther Methods Clin Dev. 2018 Nov 1;11:167-179. doi: 10.1016/j.omtm.2018.10.014. eCollection 2018 Dec 14. Mol Ther Methods Clin Dev. 2018. PMID: 30533448 Free PMC article.
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID.
Charrier S, Lagresle-Peyrou C, Poletti V, Rothe M, Cédrone G, Gjata B, Mavilio F, Fischer A, Schambach A, de Villartay JP, Cavazzana M, Hacein-Bey-Abina S, Galy A. Charrier S, et al. Among authors: mavilio f. Mol Ther Methods Clin Dev. 2019 Sep 13;15:232-245. doi: 10.1016/j.omtm.2019.08.014. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31720302 Free PMC article.
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: mavilio f. 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.
Gene Therapy for Hemoglobinopathies.
Cavazzana M, Mavilio F. Cavazzana M, et al. Among authors: mavilio f. Hum Gene Ther. 2018 Oct;29(10):1106-1113. doi: 10.1089/hum.2018.122. Hum Gene Ther. 2018. PMID: 30200783 Free PMC article. Review.
196 results