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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.
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.
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.
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: paulis m. Sci Transl Med. 2024 Feb 7;16(733):eadh8162. doi: 10.1126/scitranslmed.adh8162. Epub 2024 Feb 7. Sci Transl Med. 2024. PMID: 38324638
The K219T-Lamin mutation induces conduction defects through epigenetic inhibition of SCN5A in human cardiac laminopathy.
Salvarani N, Crasto S, Miragoli M, Bertero A, Paulis M, Kunderfranco P, Serio S, Forni A, Lucarelli C, Dal Ferro M, Larcher V, Sinagra G, Vezzoni P, Murry CE, Faggian G, Condorelli G, Di Pasquale E. Salvarani N, et al. Among authors: paulis m. Nat Commun. 2019 May 22;10(1):2267. doi: 10.1038/s41467-019-09929-w. Nat Commun. 2019. PMID: 31118417 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.
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.
83 results