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1,769 results

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Page 1
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
Castiello MC, Brandas C, Ferrari S, Porcellini S, Sacchetti N, Canarutto D, Draghici E, Merelli I, Barcella M, Pelosi G, Vavassori V, Varesi A, Jacob A, Scala S, Basso Ricci L, Paulis M, Strina D, Di Verniere M, Sergi Sergi L, Serafini M, Holland SM, Bergerson JRE, De Ravin SS, Malech HL, Pala F, Bosticardo M, Brombin C, Cugnata F, Calzoni E, Crooks GM, Notarangelo LD, Genovese P, Naldini L, Villa A. Castiello MC, et al. Among authors: villa a. Sci Transl Med. 2024 Feb 7;16(733):eadh8162. doi: 10.1126/scitranslmed.adh8162. Epub 2024 Feb 7. Sci Transl Med. 2024. PMID: 38324638
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1.
Schiroli G, Ferrari S, Conway A, Jacob A, Capo V, Albano L, Plati T, Castiello MC, Sanvito F, Gennery AR, Bovolenta C, Palchaudhuri R, Scadden DT, Holmes MC, Villa A, Sitia G, Lombardo A, Genovese P, Naldini L. Schiroli G, et al. Among authors: villa a. Sci Transl Med. 2017 Oct 11;9(411):eaan0820. doi: 10.1126/scitranslmed.aan0820. Sci Transl Med. 2017. PMID: 29021165
Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome.
Vavassori V, Mercuri E, Marcovecchio GE, Castiello MC, Schiroli G, Albano L, Margulies C, Buquicchio F, Fontana E, Beretta S, Merelli I, Cappelleri A, Rancoita PM, Lougaris V, Plebani A, Kanariou M, Lankester A, Ferrua F, Scanziani E, Cotta-Ramusino C, Villa A, Naldini L, Genovese P. Vavassori V, et al. Among authors: villa a. EMBO Mol Med. 2021 Mar 5;13(3):e13545. doi: 10.15252/emmm.202013545. Epub 2021 Jan 21. EMBO Mol Med. 2021. PMID: 33475257 Free PMC article.
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells.
Ferrari S, Jacob A, Cesana D, Laugel M, Beretta S, Varesi A, Unali G, Conti A, Canarutto D, Albano L, Calabria A, Vavassori V, Cipriani C, Castiello MC, Esposito S, Brombin C, Cugnata F, Adjali O, Ayuso E, Merelli I, Villa A, Di Micco R, Kajaste-Rudnitski A, Montini E, Penaud-Budloo M, Naldini L. Ferrari S, et al. Among authors: villa a. Cell Stem Cell. 2022 Oct 6;29(10):1428-1444.e9. doi: 10.1016/j.stem.2022.09.001. Cell Stem Cell. 2022. PMID: 36206730 Free PMC article.
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.
Aiuti A, Biasco L, Scaramuzza S, Ferrua F, Cicalese MP, Baricordi C, Dionisio F, Calabria A, Giannelli S, Castiello MC, Bosticardo M, Evangelio C, Assanelli A, Casiraghi M, Di Nunzio S, Callegaro L, Benati C, Rizzardi P, Pellin D, Di Serio C, Schmidt M, Von Kalle C, Gardner J, Mehta N, Neduva V, Dow DJ, Galy A, Miniero R, Finocchi A, Metin A, Banerjee PP, Orange JS, Galimberti S, Valsecchi MG, Biffi A, Montini E, Villa A, Ciceri F, Roncarolo MG, Naldini L. Aiuti A, et al. Among authors: villa a. Science. 2013 Aug 23;341(6148):1233151. doi: 10.1126/science.1233151. Epub 2013 Jul 11. Science. 2013. PMID: 23845947 Free PMC article. Clinical Trial.
B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome.
Castiello MC, Scaramuzza S, Pala F, Ferrua F, Uva P, Brigida I, Sereni L, van der Burg M, Ottaviano G, Albert MH, Grazia Roncarolo M, Naldini L, Aiuti A, Villa A, Bosticardo M. Castiello MC, et al. Among authors: villa a. J Allergy Clin Immunol. 2015 Sep;136(3):692-702.e2. doi: 10.1016/j.jaci.2015.01.035. Epub 2015 Mar 16. J Allergy Clin Immunol. 2015. PMID: 25792466 Free PMC article. Clinical Trial.
1,769 results