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Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo.
Asperti C, Canarutto D, Porcellini S, Sanvito F, Cecere F, Vavassori V, Ferrari S, Rovelli E, Albano L, Jacob A, Sergi Sergi L, Montaldo E, Ferrua F, González-Granado LI, Lougaris V, Badolato R, Finocchi A, Villa A, Radrizzani M, Naldini L. Asperti C, et al. Among authors: canarutto d. Mol Ther Methods Clin Dev. 2023 Aug 23;30:546-557. doi: 10.1016/j.omtm.2023.08.020. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693944 Free PMC article.
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.
Ferrua F, Cicalese MP, Galimberti S, Giannelli S, Dionisio F, Barzaghi F, Migliavacca M, Bernardo ME, Calbi V, Assanelli AA, Facchini M, Fossati C, Albertazzi E, Scaramuzza S, Brigida I, Scala S, Basso-Ricci L, Pajno R, Casiraghi M, Canarutto D, Salerio FA, Albert MH, Bartoli A, Wolf HM, Fiori R, Silvani P, Gattillo S, Villa A, Biasco L, Dott C, Culme-Seymour EJ, van Rossem K, Atkinson G, Valsecchi MG, Roncarolo MG, Ciceri F, Naldini L, Aiuti A. Ferrua F, et al. Among authors: canarutto d. Lancet Haematol. 2019 May;6(5):e239-e253. doi: 10.1016/S2352-3026(19)30021-3. Epub 2019 Apr 10. Lancet Haematol. 2019. PMID: 30981783 Free PMC article. Clinical Trial.
Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy.
Tucci F, Frittoli M, Barzaghi F, Calbi V, Migliavacca M, Ferrua F, Fumagalli F, Lorioli L, Castagnaro L, Facchini M, Fossati C, Zancan S, Massariello P, Manfredini M, Consiglieri G, Canarutto D, Recupero S, Calzatini F, Casiraghi M, Darin S, Antonioli G, Miniero R, Fiori R, Silvani P, Zambelli M, Marktel S, Gattillo S, Milani R, Santoleri L, Ciceri F, Biffi A, Cicalese MP, Bernardo ME, Aiuti A. Tucci F, et al. Among authors: canarutto d. Bone Marrow Transplant. 2019 Dec;54(12):1995-2003. doi: 10.1038/s41409-019-0573-6. Epub 2019 May 31. Bone Marrow Transplant. 2019. PMID: 31150018 Free PMC article.
Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series.
Canarutto D, Tucci F, Gattillo S, Zambelli M, Calbi V, Gentner B, Ferrua F, Marktel S, Migliavacca M, Barzaghi F, Consiglieri G, Gallo V, Fumagalli F, Massariello P, Parisi C, Viarengo G, Albertazzi E, Silvani P, Milani R, Santoleri L, Ciceri F, Cicalese MP, Bernardo ME, Aiuti A. Canarutto D, et al. Mol Ther Methods Clin Dev. 2021 Jun 1;22:76-83. doi: 10.1016/j.omtm.2021.05.013. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34485596 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: canarutto d. 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.
Case Report: Consistent disease manifestations with a staggered time course in two identical twins affected by adenosine deaminase 2 deficiency.
Barzaghi F, Cicalese MP, Zoccolillo M, Brigida I, Barcella M, Merelli I, Sartirana C, Zanussi M, Calbi V, Bernardo ME, Tucci F, Migliavacca M, Giglio F, Doglio M, Canarutto D, Ferrua F, Consiglieri G, Prunotto G, Saettini F, Bonanomi S, Rovere-Querini P, Di Colo G, Jofra T, Fousteri G, Penco F, Gattorno M, Hershfield MS, Bongiovanni L, Ponzoni M, Marktel S, Milani R, Peccatori J, Ciceri F, Mortellaro A, Aiuti A. Barzaghi F, et al. Among authors: canarutto d. Front Immunol. 2022 Sep 29;13:910021. doi: 10.3389/fimmu.2022.910021. eCollection 2022. Front Immunol. 2022. PMID: 36248833 Free PMC article.
Genotoxic effects of base and prime editing in human hematopoietic stem cells.
Fiumara M, Ferrari S, Omer-Javed A, Beretta S, Albano L, Canarutto D, Varesi A, Gaddoni C, Brombin C, Cugnata F, Zonari E, Naldini MM, Barcella M, Gentner B, Merelli I, Naldini L. Fiumara M, et al. Among authors: canarutto d. Nat Biotechnol. 2023 Sep 7. doi: 10.1038/s41587-023-01915-4. Online ahead of print. Nat Biotechnol. 2023. PMID: 37679541
26 results