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Gene Therapy for β-Hemoglobinopathies.
Cavazzana M, Antoniani C, Miccio A. Cavazzana M, et al. Among authors: miccio a. 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.
Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements.
Lattanzi A, Meneghini V, Pavani G, Amor F, Ramadier S, Felix T, Antoniani C, Masson C, Alibeu O, Lee C, Porteus MH, Bao G, Amendola M, Mavilio F, Miccio A. Lattanzi A, et al. Among authors: miccio a. Mol Ther. 2019 Jan 2;27(1):137-150. doi: 10.1016/j.ymthe.2018.10.008. Epub 2018 Oct 17. Mol Ther. 2019. PMID: 30424953 Free PMC article.
Genome editing approaches to β-hemoglobinopathies.
Brusson M, Miccio A. Brusson M, et al. Among authors: miccio a. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1. Prog Mol Biol Transl Sci. 2021. PMID: 34175041
Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease.
Ramadier S, Chalumeau A, Felix T, Othman N, Aknoun S, Casini A, Maule G, Masson C, De Cian A, Frati G, Brusson M, Concordet JP, Cavazzana M, Cereseto A, El Nemer W, Amendola M, Wattellier B, Meneghini V, Miccio A. Ramadier S, et al. Among authors: miccio a. Mol Ther. 2022 Jan 5;30(1):145-163. doi: 10.1016/j.ymthe.2021.08.019. Epub 2021 Aug 19. Mol Ther. 2022. PMID: 34418541 Free PMC article.
Recent Progress in Genome Editing for Gene Therapy Applications: The French Perspective.
Amendola M, Bedel A, Buj-Bello A, Carrara M, Concordet JP, Frati G, Gilot D, Giovannangeli C, Gutierrez-Guerrero A, Laurent M, Miccio A, Moreau-Gaudry F, Sourd C, Valton J, Verhoeyen E. Amendola M, et al. Among authors: miccio a. Hum Gene Ther. 2021 Oct;32(19-20):1059-1075. doi: 10.1089/hum.2021.191. Hum Gene Ther. 2021. PMID: 34494480 Free article. Review.
Base and Prime Editing Technologies for Blood Disorders.
Antoniou P, Miccio A, Brusson M. Antoniou P, et al. Among authors: miccio a. Front Genome Ed. 2021 Jan 28;3:618406. doi: 10.3389/fgeed.2021.618406. eCollection 2021. Front Genome Ed. 2021. PMID: 34713251 Free PMC article. Review.
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: miccio a. Nat Commun. 2022 Nov 4;13(1):6618. doi: 10.1038/s41467-022-34493-1. Nat Commun. 2022. PMID: 36333351 Free PMC article.
92 results