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39 results

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Page 1
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: porcellini s. Sci Transl Med. 2024 Feb 7;16(733):eadh8162. doi: 10.1126/scitranslmed.adh8162. Epub 2024 Feb 7. Sci Transl Med. 2024. PMID: 38324638 Free PMC article.
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: porcellini s. 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.
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: porcellini s. 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.
A p38 MAPK-ROS axis fuels proliferation stress and DNA damage during CRISPR-Cas9 gene editing in hematopoietic stem and progenitor cells.
Della Volpe L, Midena F, Vacca R, Tavella T, Alessandrini L, Farina G, Brandas C, Lo Furno E, Giannetti K, Carsana E, Naldini MM, Barcella M, Ferrari S, Beretta S, Santoro A, Porcellini S, Varesi A, Gilioli D, Conti A, Merelli I, Gentner B, Villa A, Naldini L, Di Micco R. Della Volpe L, et al. Among authors: porcellini s. Cell Rep Med. 2024 Nov 19;5(11):101823. doi: 10.1016/j.xcrm.2024.101823. Epub 2024 Nov 12. Cell Rep Med. 2024. PMID: 39536752 Free PMC article.
Wearable defibrillator to improve accuracy in selecting candidates to implantable defibrillator: A real-world experience.
Dell'Era G, Caimmi P, Spinoni EG, Battistini E, Porcellini S, De Vecchi F, Santagostino M, Ghiglieno C, Degiovanni A, Leigheb F, Kozel D, Capponi A, Patti G. Dell'Era G, et al. Among authors: porcellini s. ESC Heart Fail. 2024 Dec;11(6):3993-3999. doi: 10.1002/ehf2.14840. Epub 2024 Aug 1. ESC Heart Fail. 2024. PMID: 39091044 Free PMC article.
Characterization and Functional Analysis of CD44v6.CAR T Cells Endowed with a New Low-Affinity Nerve Growth Factor Receptor-Based Spacer.
Stornaiuolo A, Valentinis B, Sirini C, Scavullo C, Asperti C, Zhou D, Martinez De La Torre Y, Corna S, Casucci M, Porcellini S, Traversari C. Stornaiuolo A, et al. Among authors: porcellini s. Hum Gene Ther. 2021 Jul;32(13-14):744-760. doi: 10.1089/hum.2020.216. Epub 2021 May 5. Hum Gene Ther. 2021. PMID: 33554732 Free PMC article.
39 results