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Eltrombopag in children with severe aplastic anemia.
Lesmana H, Jacobs T, Boals M, Gray N, Lewis S, Ding J, Kang G, Hale M, Weiss M, Reiss U, Wang W, Wlodarski M. Lesmana H, et al. Pediatr Blood Cancer. 2021 Aug;68(8):e29066. doi: 10.1002/pbc.29066. Epub 2021 Apr 15. Pediatr Blood Cancer. 2021. PMID: 33855784
Gene Therapy and Gene Editing for β-Thalassemia.
Christakopoulos GE, Telange R, Yen J, Weiss MJ. Christakopoulos GE, et al. Among authors: weiss mj. Hematol Oncol Clin North Am. 2023 Apr;37(2):433-447. doi: 10.1016/j.hoc.2022.12.012. Hematol Oncol Clin North Am. 2023. PMID: 36907613 Free PMC article. Review.
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease.
Katta V, O'Keefe K, Li Y, Mayuranathan T, Lazzarotto CR, Wood RK, Levine RM, Powers A, Mayberry K, Manquen G, Yao Y, Zhang J, Jang Y, Nimmagadda N, Dempsey EA, Lee G, Uchida N, Cheng Y, Fazio F, Lockey T, Meagher M, Sharma A, Tisdale JF, Zhou S, Yen JS, Weiss MJ, Tsai SQ. Katta V, et al. Among authors: weiss mj. Mol Ther. 2024 Oct 2;32(10):3433-3452. doi: 10.1016/j.ymthe.2024.07.022. Epub 2024 Jul 31. Mol Ther. 2024. PMID: 39086133 Free PMC article.
Preclinical development of lentiviral vector gene therapy for Diamond-Blackfan anemia syndrome.
Bhoopalan SV, Mayuranathan T, Liu N, Mayberry K, Yao Y, Zhang J, Métais JY, Yan KK, Throm RE, Ellis SR, Ju Y, Han L, Suryaprakash S, Palmer LE, Zhou S, Yu J, Cheng Y, Yen JS, Gottschalk S, Weiss MJ. Bhoopalan SV, et al. Among authors: weiss mj. Mol Ther. 2024 Dec 12:S1525-0016(24)00819-0. doi: 10.1016/j.ymthe.2024.12.020. Online ahead of print. Mol Ther. 2024. PMID: 39673126 Free article.
710 results