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Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia.
Lidonnici MR, Paleari Y, Tiboni F, Mandelli G, Rossi C, Vezzoli M, Aprile A, Lederer CW, Ambrosi A, Chanut F, Sanvito F, Calabria A, Poletti V, Mavilio F, Montini E, Naldini L, Cristofori P, Ferrari G. Lidonnici MR, et al. Mol Ther Methods Clin Dev. 2018 Sep 13;11:9-28. doi: 10.1016/j.omtm.2018.09.001. eCollection 2018 Dec 14. Mol Ther Methods Clin Dev. 2018. PMID: 30320151 Free PMC article.
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia.
Marktel S, Scaramuzza S, Cicalese MP, Giglio F, Galimberti S, Lidonnici MR, Calbi V, Assanelli A, Bernardo ME, Rossi C, Calabria A, Milani R, Gattillo S, Benedicenti F, Spinozzi G, Aprile A, Bergami A, Casiraghi M, Consiglieri G, Masera N, D'Angelo E, Mirra N, Origa R, Tartaglione I, Perrotta S, Winter R, Coppola M, Viarengo G, Santoleri L, Graziadei G, Gabaldo M, Valsecchi MG, Montini E, Naldini L, Cappellini MD, Ciceri F, Aiuti A, Ferrari G. Marktel S, et al. Among authors: lidonnici mr. Nat Med. 2019 Feb;25(2):234-241. doi: 10.1038/s41591-018-0301-6. Epub 2019 Jan 21. Nat Med. 2019. PMID: 30664781 Clinical Trial.
Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy.
Zonari E, Desantis G, Petrillo C, Boccalatte FE, Lidonnici MR, Kajaste-Rudnitski A, Aiuti A, Ferrari G, Naldini L, Gentner B. Zonari E, et al. Among authors: lidonnici mr. Stem Cell Reports. 2017 Apr 11;8(4):977-990. doi: 10.1016/j.stemcr.2017.02.010. Epub 2017 Mar 16. Stem Cell Reports. 2017. PMID: 28330619 Free PMC article.
Plerixafor and G-CSF combination mobilizes hematopoietic stem and progenitors cells with a distinct transcriptional profile and a reduced in vivo homing capacity compared to plerixafor alone.
Lidonnici MR, Aprile A, Frittoli MC, Mandelli G, Paleari Y, Spinelli A, Gentner B, Zambelli M, Parisi C, Bellio L, Cassinerio E, Zanaboni L, Cappellini MD, Ciceri F, Marktel S, Ferrari G. Lidonnici MR, et al. Haematologica. 2017 Apr;102(4):e120-e124. doi: 10.3324/haematol.2016.154740. Epub 2016 Dec 29. Haematologica. 2017. PMID: 28034992 Free PMC article. No abstract available.
29 results