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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.
Sickle Cell Disease: From Genetics to Curative Approaches.
Hardouin G, Magrin E, Corsia A, Cavazzana M, Miccio A, Semeraro M. Hardouin G, et al. Among authors: miccio a. Annu Rev Genomics Hum Genet. 2023 Aug 25;24:255-275. doi: 10.1146/annurev-genom-120122-081037. Annu Rev Genomics Hum Genet. 2023. PMID: 37624668 Free article. Review.
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.
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: miccio a. Sci Adv. 2020 Feb 12;6(7):eaay9392. doi: 10.1126/sciadv.aay9392. Print 2020 Feb. Sci Adv. 2020. PMID: 32917636 Free PMC article.
Fetal hemoglobin rescues ineffective erythropoiesis in sickle cell disease.
El Hoss S, Cochet S, Godard A, Yan H, Dussiot M, Frati G, Boutonnat-Faucher B, Laurance S, Renaud O, Joseph L, Miccio A, Brousse V, Narla M, El Nemer W. El Hoss S, et al. Among authors: miccio a. Haematologica. 2021 Oct 1;106(10):2707-2719. doi: 10.3324/haematol.2020.265462. Haematologica. 2021. PMID: 32855279 Free PMC article.
Editorial: Mutation-Specific Gene Editing for Blood Disorders.
Lederer CW, Genovese P, Miccio A, Philipsen S. Lederer CW, et al. Among authors: miccio a. Front Genome Ed. 2021 Sep 29;3:761771. doi: 10.3389/fgeed.2021.761771. eCollection 2021. Front Genome Ed. 2021. PMID: 34713270 Free PMC article. No abstract available.
92 results