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In vivo hematopoietic stem cell modification by mRNA delivery.
Breda L, Papp TE, Triebwasser MP, Yadegari A, Fedorky MT, Tanaka N, Abdulmalik O, Pavani G, Wang Y, Grupp SA, Chou ST, Ni H, Mui BL, Tam YK, Weissman D, Rivella S, Parhiz H. Breda L, et al. Among authors: abdulmalik o. Science. 2023 Jul 28;381(6656):436-443. doi: 10.1126/science.ade6967. Epub 2023 Jul 27. Science. 2023. PMID: 37499029
Domain-focused CRISPR screen identifies HRI as a fetal hemoglobin regulator in human erythroid cells.
Grevet JD, Lan X, Hamagami N, Edwards CR, Sankaranarayanan L, Ji X, Bhardwaj SK, Face CJ, Posocco DF, Abdulmalik O, Keller CA, Giardine B, Sidoli S, Garcia BA, Chou ST, Liebhaber SA, Hardison RC, Shi J, Blobel GA. Grevet JD, et al. Among authors: abdulmalik o. Science. 2018 Jul 20;361(6399):285-290. doi: 10.1126/science.aao0932. Science. 2018. PMID: 30026227 Free PMC article.
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: abdulmalik o. Haematologica. 2019 May 1;106(5):1433-1442. doi: 10.3324/haematol.2019.226852. Haematologica. 2019. PMID: 32439726 Free PMC article.
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: abdulmalik o. 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: abdulmalik o. Haematologica. 2021 Oct 1;106(10):2740-2745. doi: 10.3324/haematol.2020.276634. Haematologica. 2021. PMID: 34047176 Free PMC article. No abstract available.
Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells.
Qin K, Huang P, Feng R, Keller CA, Peslak SA, Khandros E, Saari MS, Lan X, Mayuranathan T, Doerfler PA, Abdulmalik O, Giardine B, Chou ST, Shi J, Hardison RC, Weiss MJ, Blobel GA. Qin K, et al. Among authors: abdulmalik o. Nat Genet. 2022 Jun;54(6):874-884. doi: 10.1038/s41588-022-01076-1. Epub 2022 May 26. Nat Genet. 2022. PMID: 35618846 Free PMC article.
HIC2 controls developmental hemoglobin switching by repressing BCL11A transcription.
Huang P, Peslak SA, Ren R, Khandros E, Qin K, Keller CA, Giardine B, Bell HW, Lan X, Sharma M, Horton JR, Abdulmalik O, Chou ST, Shi J, Crossley M, Hardison RC, Cheng X, Blobel GA. Huang P, et al. Among authors: abdulmalik o. Nat Genet. 2022 Sep;54(9):1417-1426. doi: 10.1038/s41588-022-01152-6. Epub 2022 Aug 8. Nat Genet. 2022. PMID: 35941187 Free PMC article.
83 results