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Page 1
High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing.
Plasschaert RN, DeAndrade MP, Hull F, Nguyen Q, Peterson T, Yan A, Loperfido M, Baricordi C, Barbarossa L, Yoon JK, Dogan Y, Unnisa Z, Schindler JW, van Til NP, Biasco L, Mason C. Plasschaert RN, et al. Among authors: loperfido m. Mol Ther. 2022 Oct 5;30(10):3209-3225. doi: 10.1016/j.ymthe.2022.05.022. Epub 2022 May 25. Mol Ther. 2022. PMID: 35614857 Free PMC article.
Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome.
Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Hernandez RJ, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae CC, Vicenzi E, Finocchi A, Bredius R, Bosticardo M, Schmidt M, von Kalle C, Montini E, Biffi A, Roncarolo MG, Naldini L, Villa A, Aiuti A. Scaramuzza S, et al. Among authors: loperfido m. Mol Ther. 2013 Jan;21(1):175-84. doi: 10.1038/mt.2012.23. Epub 2012 Feb 28. Mol Ther. 2013. PMID: 22371846 Free PMC article.
piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.
Loperfido M, Jarmin S, Dastidar S, Di Matteo M, Perini I, Moore M, Nair N, Samara-Kuko E, Athanasopoulos T, Tedesco FS, Dickson G, Sampaolesi M, VandenDriessche T, Chuah MK. Loperfido M, et al. Nucleic Acids Res. 2016 Jan 29;44(2):744-60. doi: 10.1093/nar/gkv1464. Epub 2015 Dec 17. Nucleic Acids Res. 2016. PMID: 26682797 Free PMC article.
Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases.
Daboussi F, Zaslavskiy M, Poirot L, Loperfido M, Gouble A, Guyot V, Leduc S, Galetto R, Grizot S, Oficjalska D, Perez C, Delacôte F, Dupuy A, Chion-Sotinel I, Le Clerre D, Lebuhotel C, Danos O, Lemaire F, Oussedik K, Cédrone F, Epinat JC, Smith J, Yáñez-Muñoz RJ, Dickson G, Popplewell L, Koo T, VandenDriessche T, Chuah MK, Duclert A, Duchateau P, Pâques F. Daboussi F, et al. Among authors: loperfido m. Nucleic Acids Res. 2012 Jul;40(13):6367-79. doi: 10.1093/nar/gks268. Epub 2012 Mar 29. Nucleic Acids Res. 2012. PMID: 22467209 Free PMC article.
Next-generation muscle-directed gene therapy by in silico vector design.
Sarcar S, Tulalamba W, Rincon MY, Tipanee J, Pham HQ, Evens H, Boon D, Samara-Kuko E, Keyaerts M, Loperfido M, Berardi E, Jarmin S, In't Veld P, Dickson G, Lahoutte T, Sampaolesi M, De Bleser P, VandenDriessche T, Chuah MK. Sarcar S, et al. Among authors: loperfido m. Nat Commun. 2019 Jan 30;10(1):492. doi: 10.1038/s41467-018-08283-7. Nat Commun. 2019. PMID: 30700722 Free PMC article.
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.
Yoon JK, Schindler JW, Loperfido M, Baricordi C, DeAndrade MP, Jacobs ME, Treleaven C, Plasschaert RN, Yan A, Barese CN, Dogan Y, Chen VP, Fiorini C, Hull F, Barbarossa L, Unnisa Z, Ivanov D, Kutner RH, Guda S, Oborski C, Maiwald T, Michaud V, Rothe M, Schambach A, Pfeifer R, Mason C, Biasco L, van Til NP. Yoon JK, et al. Among authors: loperfido m. Mol Ther. 2024 Nov 6;32(11):3847-3864. doi: 10.1016/j.ymthe.2024.09.024. Epub 2024 Sep 17. Mol Ther. 2024. PMID: 39295144
17 results