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Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System.
Pontesilli S, Baldoli C, Rosa PAD, Cattoni A, Bernardo ME, Meregalli P, Gasperini S, Motta S, Fumagalli F, Tucci F, Baciga F, Consiglieri G, Canonico F, De Lorenzo P, Chiapparini L, Gentner B, Aiuti A, Biondi A, Rovelli A, Parini R. Pontesilli S, et al. Among authors: gentner b. J Pediatr. 2022 Jan;240:297-301.e5. doi: 10.1016/j.jpeds.2021.09.020. Epub 2021 Sep 20. J Pediatr. 2022. PMID: 34547335
Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.
Chiriaco M, Farinelli G, Capo V, Zonari E, Scaramuzza S, Di Matteo G, Sergi LS, Migliavacca M, Hernandez RJ, Bombelli F, Giorda E, Kajaste-Rudnitski A, Trono D, Grez M, Rossi P, Finocchi A, Naldini L, Gentner B, Aiuti A. Chiriaco M, et al. Among authors: gentner b. Mol Ther. 2014 Aug;22(8):1472-1483. doi: 10.1038/mt.2014.87. Epub 2014 May 29. Mol Ther. 2014. PMID: 24869932 Free PMC article.
Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy.
Zonari E, Desantis G, Petrillo C, Boccalatte FE, Lidonnici MR, Kajaste-Rudnitski A, Aiuti A, Ferrari G, Naldini L, Gentner B. Zonari E, et al. Among authors: gentner b. Stem Cell Reports. 2017 Apr 11;8(4):977-990. doi: 10.1016/j.stemcr.2017.02.010. Epub 2017 Mar 16. Stem Cell Reports. 2017. PMID: 28330619 Free PMC article.
Neonatal umbilical cord blood transplantation halts skeletal disease progression in the murine model of MPS-I.
Azario I, Pievani A, Del Priore F, Antolini L, Santi L, Corsi A, Cardinale L, Sawamoto K, Kubaski F, Gentner B, Bernardo ME, Valsecchi MG, Riminucci M, Tomatsu S, Aiuti A, Biondi A, Serafini M. Azario I, et al. Among authors: gentner b. Sci Rep. 2017 Aug 25;7(1):9473. doi: 10.1038/s41598-017-09958-9. Sci Rep. 2017. PMID: 28842642 Free PMC article.
Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches.
Fraldi A, Serafini M, Sorrentino NC, Gentner B, Aiuti A, Bernardo ME. Fraldi A, et al. Among authors: gentner b. Ital J Pediatr. 2018 Nov 16;44(Suppl 2):130. doi: 10.1186/s13052-018-0565-y. Ital J Pediatr. 2018. PMID: 30442177 Free PMC article. Review.
Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease.
Squeri G, Passerini L, Ferro F, Laudisa C, Tomasoni D, Deodato F, Donati MA, Gasperini S, Aiuti A, Bernardo ME, Gentner B, Naldini L, Annoni A, Biffi A, Gregori S. Squeri G, et al. Among authors: gentner b. Mol Ther. 2019 Jul 3;27(7):1215-1227. doi: 10.1016/j.ymthe.2019.04.014. Epub 2019 Apr 19. Mol Ther. 2019. PMID: 31060789 Free PMC article.
Expanded circulating hematopoietic stem/progenitor cells as novel cell source for the treatment of TCIRG1 osteopetrosis.
Capo V, Penna S, Merelli I, Barcella M, Scala S, Basso-Ricci L, Draghici E, Palagano E, Zonari E, Desantis G, Uva P, Cusano R, Sergi Sergi L, Crisafulli L, Moshous D, Stepensky P, Drabko K, Kaya Z, Unal E, Gezdirici A, Menna G, Serafini M, Aiuti A, Locatelli SL, Carlo-Stella C, Schulz AS, Ficara F, Sobacchi C, Gentner B, Villa A. Capo V, et al. Among authors: gentner b. Haematologica. 2021 Jan 1;106(1):74-86. doi: 10.3324/haematol.2019.238261. Haematologica. 2021. PMID: 31949009 Free PMC article.
Pro-inflammatory cytokines favor the emergence of ETV6-RUNX1-positive pre-leukemic cells in a model of mesenchymal niche.
Beneforti L, Dander E, Bresolin S, Bueno C, Acunzo D, Bertagna M, Ford A, Gentner B, Kronnie GT, Vergani P, Menéndez P, Biondi A, D'Amico G, Palmi C, Cazzaniga G. Beneforti L, et al. Among authors: gentner b. Br J Haematol. 2020 Jul;190(2):262-273. doi: 10.1111/bjh.16523. Epub 2020 Mar 2. Br J Haematol. 2020. PMID: 32118299 Free article.
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy.
Jofra Hernández R, Calabria A, Sanvito F, De Mattia F, Farinelli G, Scala S, Visigalli I, Carriglio N, De Simone M, Vezzoli M, Cecere F, Migliavacca M, Basso-Ricci L, Omrani M, Benedicenti F, Norata R, Rancoita PMV, Di Serio C, Albertini P, Cristofori P, Naldini L, Gentner B, Montini E, Aiuti A, Mortellaro A. Jofra Hernández R, et al. Among authors: gentner b. Mol Ther. 2021 Jan 6;29(1):86-102. doi: 10.1016/j.ymthe.2020.09.030. Epub 2020 Sep 23. Mol Ther. 2021. PMID: 33010230 Free PMC article.
74 results