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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: zhang j. 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: zhang j. 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.
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: zhang j. 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.
Activation of γ-globin expression by hypoxia-inducible factor 1α.
Feng R, Mayuranathan T, Huang P, Doerfler PA, Li Y, Yao Y, Zhang J, Palmer LE, Mayberry K, Christakopoulos GE, Xu P, Li C, Cheng Y, Blobel GA, Simon MC, Weiss MJ. Feng R, et al. Among authors: zhang j. Nature. 2022 Oct;610(7933):783-790. doi: 10.1038/s41586-022-05312-w. Epub 2022 Oct 12. Nature. 2022. PMID: 36224385 Free PMC article.
The autophagy-activating kinase ULK1 mediates clearance of free α-globin in β-thalassemia.
Lechauve C, Keith J, Khandros E, Fowler S, Mayberry K, Freiwan A, Thom CS, Delbini P, Romero EB, Zhang J, Motta I, Tillman H, Cappellini MD, Kundu M, Weiss MJ. Lechauve C, et al. Among authors: zhang j. Sci Transl Med. 2019 Aug 21;11(506):eaav4881. doi: 10.1126/scitranslmed.aav4881. Sci Transl Med. 2019. PMID: 31434755 Free PMC article.
178,088 results
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