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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.
Improvements in haemolysis and indicators of erythrocyte survival do not correlate with acute vaso-occlusive crises in patients with sickle cell disease: a phase III randomized, placebo-controlled, double-blind study of the Gardos channel blocker senicapoc (ICA-17043).
Ataga KI, Reid M, Ballas SK, Yasin Z, Bigelow C, James LS, Smith WR, Galacteros F, Kutlar A, Hull JH, Stocker JW; ICA-17043-10 Study Investigators. Ataga KI, et al. Br J Haematol. 2011 Apr;153(1):92-104. doi: 10.1111/j.1365-2141.2010.08520.x. Epub 2011 Feb 17. Br J Haematol. 2011. PMID: 21323872 Free article. Clinical Trial.
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. 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. Haematologica. 2021 Oct 1;106(10):2740-2745. doi: 10.3324/haematol.2020.276634. Haematologica. 2021. PMID: 34047176 Free PMC article. No abstract available.
Consensus definition of essential, optimal, and suggested components of a pediatric sickle cell disease center.
Hulbert ML, Manwani D, Meier ER, Alvarez OA, Brown RC, Callaghan MU, Campbell AD, Coates TD, Frei-Jones MJ, Hankins JS, Heeney MM, Hsu LL, Lebensburger JD, Quinn CT, Shah N, Smith-Whitley K, Thornburg C, Kanter J. Hulbert ML, et al. Pediatr Blood Cancer. 2023 Jan;70(1):e29961. doi: 10.1002/pbc.29961. Epub 2022 Sep 12. Pediatr Blood Cancer. 2023. PMID: 36094289
Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment (HABIT) efficacy trial: Community health worker support may increase hydroxyurea adherence of youth with sickle cell disease.
Green NS, Manwani D, Aygun B, Appiah-Kubi A, Smith-Whitley K, Castillo Y, Soriano L, Jia H, Smaldone AM. Green NS, et al. Among authors: smith whitley k. Pediatr Blood Cancer. 2024 Apr;71(4):e30878. doi: 10.1002/pbc.30878. Epub 2024 Feb 6. Pediatr Blood Cancer. 2024. PMID: 38321562
116 results