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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: guzikowski 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: guzikowski 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.