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Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial.
Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Tréluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poirée M, Monpoux F, Touraine P, Brouzes C, de Montalembert M, Payen E, Six E, Ribeil JA, Miccio A, Bartolucci P, Leboulch P, Cavazzana M. Magrin E, et al. Among authors: miccio a. Nat Med. 2022 Jan;28(1):81-88. doi: 10.1038/s41591-021-01650-w. Epub 2022 Jan 24. Nat Med. 2022. PMID: 35075288 Clinical Trial.
Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi.
Cavazza A, Hendel A, Bak RO, Rio P, Güell M, Lainšček D, Arechavala-Gomeza V, Peng L, Hapil FZ, Harvey J, Ortega FG, Gonzalez-Martinez C, Lederer CW, Mikkelsen K, Gasiunas G, Kalter N, Gonçalves MAFV, Petersen J, Garanto A, Montoliu L, Maresca M, Seemann SE, Gorodkin J, Mazini L, Sanchez R, Rodriguez-Madoz JR, Maldonado-Pérez N, Laura T, Schmueck-Henneresse M, Maccalli C, Grünewald J, Carmona G, Kachamakova-Trojanowska N, Miccio A, Martin F, Turchiano G, Cathomen T, Luo Y, Tsai SQ, Benabdellah K; COST Action CA21113. Cavazza A, et al. Among authors: miccio a. Mol Ther Nucleic Acids. 2023 Oct 29;34:102066. doi: 10.1016/j.omtn.2023.102066. eCollection 2023 Dec 12. Mol Ther Nucleic Acids. 2023. PMID: 38034032 Free PMC article. Review.
An optimized SpCas9 high-fidelity variant for direct protein delivery.
Pedrazzoli E, Bianchi A, Umbach A, Amistadi S, Brusson M, Frati G, Ciciani M, Badowska KA, Arosio D, Miccio A, Cereseto A, Casini A. Pedrazzoli E, et al. Among authors: miccio a. Mol Ther. 2023 Jul 5;31(7):2257-2265. doi: 10.1016/j.ymthe.2023.03.007. Epub 2023 Mar 10. Mol Ther. 2023. PMID: 36905119 Free article.
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.
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: miccio a. 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: miccio a. 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.
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: miccio a. 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.
92 results