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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
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.
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.
Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4+ T-cells for the treatment of Hyper IgM1.
Canarutto D, Asperti C, Vavassori V, Porcellini S, Rovelli E, Paulis M, Ferrari S, Varesi A, Fiumara M, Jacob A, Sergi Sergi L, Visigalli I, Ferrua F, González-Granado LI, Lougaris V, Finocchi A, Villa A, Radrizzani M, Naldini L. Canarutto D, et al. Among authors: paulis m. EMBO J. 2023 Dec 1;42(23):e114188. doi: 10.15252/embj.2023114188. Epub 2023 Nov 2. EMBO J. 2023. PMID: 37916874 Free PMC article.
Chromosome transfer via cell fusion.
Paulis M. Paulis M. Methods Mol Biol. 2011;738:57-67. doi: 10.1007/978-1-61779-099-7_4. Methods Mol Biol. 2011. PMID: 21431719
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.
84 results