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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. 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: dong a. 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.
Differences in the effect of repetitive transcranial magnetic stimulation and median nerve electrical stimulation in patients with prolonged disorders of consciousness after intracerebral hemorrhage: a randomized controlled trial protocol.
Zhao G, Fang M, Han S, Peng X, Dong A. Zhao G, et al. Among authors: dong a. Front Neurol. 2024 Nov 28;15:1511767. doi: 10.3389/fneur.2024.1511767. eCollection 2024. Front Neurol. 2024. PMID: 39669104 Free PMC article.
1,426 results