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Page 1
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: yen js. 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.
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: yen js. 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.
Gene Therapy and Gene Editing for β-Thalassemia.
Christakopoulos GE, Telange R, Yen J, Weiss MJ. Christakopoulos GE, et al. 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.
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.
Everette KA, Newby GA, Levine RM, Mayberry K, Jang Y, Mayuranathan T, Nimmagadda N, Dempsey E, Li Y, Bhoopalan SV, Liu X, Davis JR, Nelson AT, Chen PJ, Sousa AA, Cheng Y, Tisdale JF, Weiss MJ, Yen JS, Liu DR. Everette KA, et al. Among authors: yen js. Nat Biomed Eng. 2023 May;7(5):616-628. doi: 10.1038/s41551-023-01026-0. Epub 2023 Apr 17. Nat Biomed Eng. 2023. PMID: 37069266 Free PMC article.
Potent and uniform fetal hemoglobin induction via base editing.
Mayuranathan T, Newby GA, Feng R, Yao Y, Mayberry KD, Lazzarotto CR, Li Y, Levine RM, Nimmagadda N, Dempsey E, Kang G, Porter SN, Doerfler PA, Zhang J, Jang Y, Chen J, Bell HW, Crossley M, Bhoopalan SV, Sharma A, Tisdale JF, Pruett-Miller SM, Cheng Y, Tsai SQ, Liu DR, Weiss MJ, Yen JS. Mayuranathan T, et al. Among authors: yen js. Nat Genet. 2023 Jul;55(7):1210-1220. doi: 10.1038/s41588-023-01434-7. Epub 2023 Jul 3. Nat Genet. 2023. PMID: 37400614 Free PMC article.
Base editing of haematopoietic stem cells rescues sickle cell disease in mice.
Newby GA, Yen JS, Woodard KJ, Mayuranathan T, Lazzarotto CR, Li Y, Sheppard-Tillman H, Porter SN, Yao Y, Mayberry K, Everette KA, Jang Y, Podracky CJ, Thaman E, Lechauve C, Sharma A, Henderson JM, Richter MF, Zhao KT, Miller SM, Wang T, Koblan LW, McCaffrey AP, Tisdale JF, Kalfa TA, Pruett-Miller SM, Tsai SQ, Weiss MJ, Liu DR. Newby GA, et al. Among authors: yen js. Nature. 2021 Jul;595(7866):295-302. doi: 10.1038/s41586-021-03609-w. Epub 2021 Jun 2. Nature. 2021. PMID: 34079130 Free PMC article.
Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells.
Lian X, Chatterjee S, Sun Y, Dilliard SA, Moore S, Xiao Y, Bian X, Yamada K, Sung YC, Levine RM, Mayberry K, John S, Liu X, Smith C, Johnson LT, Wang X, Zhang CC, Liu DR, Newby GA, Weiss MJ, Yen JS, Siegwart DJ. Lian X, et al. Among authors: yen js. Nat Nanotechnol. 2024 Sep;19(9):1409-1417. doi: 10.1038/s41565-024-01680-8. Epub 2024 May 23. Nat Nanotechnol. 2024. PMID: 38783058
27 results