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Page 1
Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies.
Breda L, Ghiaccio V, Tanaka N, Jarocha D, Ikawa Y, Abdulmalik O, Dong A, Casu C, Raabe TD, Shan X, Danet-Desnoyers GA, Doto AM, Everett J, Bushman FD, Radaelli E, Assenmacher CA, Tarrant JC, Hoepp N, Kurita R, Nakamura Y, Guzikowski V, Smith-Whitley K, Kwiatkowski JL, Rivella S. Breda L, et al. Among authors: ghiaccio v. Mol Ther. 2021 Apr 7;29(4):1625-1638. doi: 10.1016/j.ymthe.2020.12.036. Epub 2021 Jan 29. Mol Ther. 2021. PMID: 33515514 Free PMC article.
Inclusion of a short hairpin RNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin.
Pires Lourenco S, Jarocha D, Ghiaccio V, Guerra A, Abdulmalik O, La P, Zezulin A, Smith-Whitley K, Kwiatkowski JL, Guzikowski V, Nakamura Y, Raabe T, Breda L, Rivella S. Pires Lourenco S, et al. Among authors: ghiaccio v. Haematologica. 2021 Oct 1;106(10):2740-2745. doi: 10.3324/haematol.2020.276634. Haematologica. 2021. PMID: 34047176 Free PMC article. No abstract available.
2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance.
Dong A, Ghiaccio V, Motta I, Guo S, Peralta R, Freier SM, Watt A, Damle S, Ikawa Y, Jarocha D, Chappell M, Stephanou C, Delbini P, Chen C, Christou S, Kleanthous M, Smith-Whitley K, Manwani D, Casu C, Abdulmalik O, Cappellini MD, Rivella S, Breda L. Dong A, et al. Among authors: ghiaccio v. Haematologica. 2019 May 1;106(5):1433-1442. doi: 10.3324/haematol.2019.226852. Haematologica. 2019. PMID: 32439726 Free PMC article.
Tumor Microenvironment Remodeling by 4-Methylumbelliferone Boosts the Antitumor Effect of Combined Immunotherapy in Murine Colorectal Carcinoma.
Malvicini M, Fiore E, Ghiaccio V, Piccioni F, Rizzo M, Olmedo Bonadeo L, García M, Rodríguez M, Bayo J, Peixoto E, Atorrasagasti C, Alaniz L, Aquino J, Matar P, Mazzolini G. Malvicini M, et al. Among authors: ghiaccio v. Mol Ther. 2015 Sep;23(9):1444-55. doi: 10.1038/mt.2015.112. Epub 2015 Jun 24. Mol Ther. 2015. PMID: 26105158 Free PMC article.
Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy.
Chappell ME, Breda L, Tricoli L, Guerra A, Jarocha D, Castruccio Castracani C, Papp TE, Tanaka N, Hamilton N, Triebwasser MP, Ghiaccio V, Fedorky MT, Gollomp KL, Bochenek V, Roche AM, Everett JK, Cook EJ, Bushman FD, Teawtrakul N, Glentis S, Kattamis A, Mui BL, Tam YK, Weissman D, Abdulmalik O, Parhiz H, Rivella S. Chappell ME, et al. Among authors: ghiaccio v. Blood. 2024 Oct 10;144(15):1633-1645. doi: 10.1182/blood.2023023349. Blood. 2024. PMID: 38949981