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Targeted gene therapy and cell reprogramming in Fanconi anemia.
Rio P, Baños R, Lombardo A, Quintana-Bustamante O, Alvarez L, Garate Z, Genovese P, Almarza E, Valeri A, Díez B, Navarro S, Torres Y, Trujillo JP, Murillas R, Segovia JC, Samper E, Surralles J, Gregory PD, Holmes MC, Naldini L, Bueren JA. Rio P, et al. Among authors: holmes mc. EMBO Mol Med. 2014 Jun;6(6):835-48. doi: 10.15252/emmm.201303374. Epub 2014 Apr 6. EMBO Mol Med. 2014. PMID: 24859981 Free PMC article.
Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients.
Diez B, Genovese P, Roman-Rodriguez FJ, Alvarez L, Schiroli G, Ugalde L, Rodriguez-Perales S, Sevilla J, Diaz de Heredia C, Holmes MC, Lombardo A, Naldini L, Bueren JA, Rio P. Diez B, et al. Among authors: holmes mc. EMBO Mol Med. 2017 Nov;9(11):1574-1588. doi: 10.15252/emmm.201707540. EMBO Mol Med. 2017. PMID: 28899930 Free PMC article.
Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer.
Provasi E, Genovese P, Lombardo A, Magnani Z, Liu PQ, Reik A, Chu V, Paschon DE, Zhang L, Kuball J, Camisa B, Bondanza A, Casorati G, Ponzoni M, Ciceri F, Bordignon C, Greenberg PD, Holmes MC, Gregory PD, Naldini L, Bonini C. Provasi E, et al. Among authors: holmes mc. Nat Med. 2012 May;18(5):807-815. doi: 10.1038/nm.2700. Nat Med. 2012. PMID: 22466705 Free PMC article.
Targeted genome editing in human repopulating haematopoietic stem cells.
Genovese P, Schiroli G, Escobar G, Tomaso TD, Firrito C, Calabria A, Moi D, Mazzieri R, Bonini C, Holmes MC, Gregory PD, van der Burg M, Gentner B, Montini E, Lombardo A, Naldini L. Genovese P, et al. Among authors: holmes mc. Nature. 2014 Jun 12;510(7504):235-240. doi: 10.1038/nature13420. Epub 2014 May 28. Nature. 2014. PMID: 24870228 Free PMC article.
Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects.
Laskowski TJ, Van Caeneghem Y, Pourebrahim R, Ma C, Ni Z, Garate Z, Crane AM, Li XS, Liao W, Gonzalez-Garay M, Segovia JC, Paschon DE, Rebar EJ, Holmes MC, Kaufman D, Vandekerckhove B, Davis BR. Laskowski TJ, et al. Among authors: holmes mc. Stem Cell Reports. 2016 Aug 9;7(2):139-48. doi: 10.1016/j.stemcr.2016.06.003. Epub 2016 Jul 7. Stem Cell Reports. 2016. PMID: 27396937 Free PMC article.
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: holmes mc. Sci Transl Med. 2017 Oct 11;9(411):eaan0820. doi: 10.1126/scitranslmed.aan0820. Sci Transl Med. 2017. PMID: 29021165
229 results