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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: paulis m. 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.
Anti-CD3ε mAb improves thymic architecture and prevents autoimmune manifestations in a mouse model of Omenn syndrome: therapeutic implications.
Marrella V, Poliani PL, Fontana E, Casati A, Maina V, Cassani B, Ficara F, Cominelli M, Schena F, Paulis M, Traggiai E, Vezzoni P, Grassi F, Villa A. Marrella V, et al. Among authors: paulis m. Blood. 2012 Aug 2;120(5):1005-14. doi: 10.1182/blood-2012-01-406827. Epub 2012 Jun 21. Blood. 2012. PMID: 22723555 Free PMC article.
Osteopetrosis rescue upon RANKL administration to Rankl(-/-) mice: a new therapy for human RANKL-dependent ARO.
Lo Iacono N, Blair HC, Poliani PL, Marrella V, Ficara F, Cassani B, Facchetti F, Fontana E, Guerrini MM, Traggiai E, Schena F, Paulis M, Mantero S, Inforzato A, Valaperta S, Pangrazio A, Crisafulli L, Maina V, Kostenuik P, Vezzoni P, Villa A, Sobacchi C. Lo Iacono N, et al. Among authors: paulis m. J Bone Miner Res. 2012 Dec;27(12):2501-10. doi: 10.1002/jbmr.1712. J Bone Miner Res. 2012. PMID: 22836362 Free article.
Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts.
Neri T, Muggeo S, Paulis M, Caldana ME, Crisafulli L, Strina D, Focarelli ML, Faggioli F, Recordati C, Scaramuzza S, Scanziani E, Mantero S, Buracchi C, Sobacchi C, Lombardo A, Naldini L, Vezzoni P, Villa A, Ficara F. Neri T, et al. Among authors: paulis m. Stem Cell Reports. 2015 Oct 13;5(4):558-68. doi: 10.1016/j.stemcr.2015.08.005. Epub 2015 Sep 3. Stem Cell Reports. 2015. PMID: 26344905 Free PMC article.
Chromosome Transplantation: A Possible Approach to Treat Human X-linked Disorders.
Paulis M, Susani L, Castelli A, Suzuki T, Hara T, Straniero L, Duga S, Strina D, Mantero S, Caldana E, Sergi LS, Villa A, Vezzoni P. Paulis M, et al. Mol Ther Methods Clin Dev. 2020 Jan 21;17:369-377. doi: 10.1016/j.omtm.2020.01.003. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32099849 Free PMC article.
Frataxin gene editing rescues Friedreich's ataxia pathology in dorsal root ganglia organoid-derived sensory neurons.
Mazzara PG, Muggeo S, Luoni M, Massimino L, Zaghi M, Valverde PT, Brusco S, Marzi MJ, Palma C, Colasante G, Iannielli A, Paulis M, Cordiglieri C, Giannelli SG, Podini P, Gellera C, Taroni F, Nicassio F, Rasponi M, Broccoli V. Mazzara PG, et al. Among authors: paulis m. Nat Commun. 2020 Aug 21;11(1):4178. doi: 10.1038/s41467-020-17954-3. Nat Commun. 2020. PMID: 32826895 Free PMC article.
Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient- and cell type-specific disease phenotypes.
Mangiameli E, Cecchele A, Morena F, Sanvito F, Matafora V, Cattaneo A, Della Volpe L, Gnani D, Paulis M, Susani L, Martino S, Di Micco R, Bachi A, Gritti A. Mangiameli E, et al. Among authors: paulis m. Stem Cell Reports. 2021 Jun 8;16(6):1478-1495. doi: 10.1016/j.stemcr.2021.04.011. Epub 2021 May 13. Stem Cell Reports. 2021. PMID: 33989519 Free PMC article.
84 results