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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: frati g. 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.
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: frati g. Sci Adv. 2020 Feb 12;6(7):eaay9392. doi: 10.1126/sciadv.aay9392. Print 2020 Feb. Sci Adv. 2020. PMID: 32917636 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: frati g. 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.
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: frati g. Haematologica. 2021 Oct 1;106(10):2707-2719. doi: 10.3324/haematol.2020.265462. Haematologica. 2021. PMID: 32855279 Free PMC article.
Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy.
Meneghini V, Frati G, Sala D, De Cicco S, Luciani M, Cavazzin C, Paulis M, Mentzen W, Morena F, Giannelli S, Sanvito F, Villa A, Bulfone A, Broccoli V, Martino S, Gritti A. Meneghini V, et al. Among authors: frati g. Stem Cells Transl Med. 2017 Feb;6(2):352-368. doi: 10.5966/sctm.2015-0414. Epub 2016 Sep 16. Stem Cells Transl Med. 2017. PMID: 28191778 Free PMC article.
DNA damage contributes to neurotoxic inflammation in Aicardi-Goutières syndrome astrocytes.
Giordano AMS, Luciani M, Gatto F, Abou Alezz M, Beghè C, Della Volpe L, Migliara A, Valsoni S, Genua M, Dzieciatkowska M, Frati G, Tahraoui-Bories J, Giliani SC, Orcesi S, Fazzi E, Ostuni R, D'Alessandro A, Di Micco R, Merelli I, Lombardo A, Reijns MAM, Gromak N, Gritti A, Kajaste-Rudnitski A. Giordano AMS, et al. Among authors: frati g. J Exp Med. 2022 Apr 4;219(4):e20211121. doi: 10.1084/jem.20211121. Epub 2022 Mar 9. J Exp Med. 2022. PMID: 35262626 Free PMC article.
446 results