Selected Publications
Everette KA, Newby GA, Levine RM, Mayberry K, Jang Y, Mayuranathan T, Nimmagadda N, Dempsey EA, Li Y, Bhoopalan SV, Liu X, Davis J, Chen P, Sousa A, Cheng Y, Tisdale JF, Weiss MJ*, Yen JS*, Liu DR*. Ex vivo prime editing reverts the sickle cell allele in hematopoietic stem cells and corrects disease phenotypes in mice. Nat. Biomed. Eng., 2023 May;7(5):616-628. PubMed Central ID: PMC10195679.
Mayuranathan T, Newby GA, Feng R, Yao Y, Mayberry K, Lazzarotto CR, Li Y, Levine RM, Nimmagadda N, Dempsey EA, 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*. Potent and uniform fetal hemoglobin induction via base editing. Nat Gen, 2023, 2023 Jul 3; PubMed ID: 37400614.
Christakopoulos GE, Telange R, Yen JS, Weiss MJ. Gene Therapy and Gene Editing for b-Thalassemia. Hematol Oncol Clin N Am 37 (2023) 433–447
Bhoopalan SV, Yen JS, Levine RM, Sharma A. Editing human hematopoietic stem cells: advances and challenges. Cytotherapy. 2022 Sep 17.
Bhoopalan SV, Yen J, Mayuranathan T, Yao Y, Mayberry K, Zhou S, Throm R, Wlodarski MW, Weiss MJ. A A novel RPS19- edited model for Diamond-Blackfan anemia reveals TP53-dependent impairment of hematopoietic stem cell activity. JCI Insights, 2023 Jan 10;8(1) PubMed Central ID: PMC9870085.
Woodard KJ, Doerfler PA, Mayberry K, Sharma A, Levine R, Yen J, Valentine V, Valentine M, Weiss MJ. Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations. Disease Models & Mechanisms, 2022 Jun 1;15(6):dmm049463.
Newby GA#, Yen J#*, 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*. Base editing of haematopoietic stem cells rescues sickle cell disease in mice. Nature. 2021 Jul;595(7866):295-302. Epub 2021 Jun 2. PMID: 34079130.
Gaudelli NM, Lam DK, Rees HA, Solá-Esteves NM, Barrera LA, Born DA, Edwards A, Gehrke JM, Lee SJ, Liquori AJ, Murray R, Packer MS, Rinaldi C, Slaymaker IM, Yen J, Young LE, Ciaramella G. Directed evolution of adenine base editors with increased activity and therapeutic application. Nat Biotechnol. 2020 Jul;38(7):892-900. Epub 2020 Apr 13. PMID: 32284586.
Yen J, Fiorino M, Liu Y, Paula S, Clarkson S, Quinn L, Tschantz WR, Klock H, Guo N, Russ C, Yu VWC, Mickanin C, Stevenson SC, Lee C, Yang Y. TRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem Cells. Sci Rep. 2018 Nov 2;8(1):16304. PMID: 30389991.
Ying H, Yen J, Wang R, Lai Y, Hsu JL, Hu Y, Cheng J. Degradable and biocompatible hydrogels bearing a hindered urea bond. Biomater Sci. 2017 Nov 21;5(12):2398-2402. PMID: 29067374.
Wang H, Wang R, Cai K, He H, Liu Y, Yen J, Wang Z, Xu M, Sun Y, Zhou X, Yin Q, Tang L, Dobrucki IT, Dobrucki LW, Chaney EJ, Boppart SA, Fan TM, Lezmi S, Chen X, Yin L, Cheng J. Selective in vivo metabolic cell-labeling-mediated cancer targeting. Nat Chem Biol. 2017 Apr;13(4):415-424. Epub 2017 Feb 13. PMID: 28192414.
Yen J, Ying H, Wang H, Yin L, Uckun F, Cheng J. CD44 Mediated Nonviral Gene Delivery into Human Embryonic Stem Cells via Hyaluronic-Acid-Coated Nanoparticles. ACS Biomater Sci Eng. 2016 Mar 14;2(3):326-335. Epub 2016 Feb 23. PMID: 33429536.
Cai K, Yen J, Yin Q, Liu Y, Song Z, Lezmi S, Zhang Y, Yang X, Helferich WG, Cheng J. Redox-Responsive Self-Assembled Chain-Shattering Polymeric Therapeutics. Biomater Sci. 2015 Jul;3(7):1061-5. PMID: 26146551.
Tang L, Yin Q, Xu Y, Zhou Q, Cai K, Yen J, Dobrucki LW, Cheng J. Bioorthogonal Oxime Ligation Mediated In Vivo Cancer Targeting. Chem Sci. 2015 Apr 1;6(4):2182-2186. PMID: 26146536.
Yen J, Yin L, Cheng J. Enhanced Non-Viral Gene Delivery to Human Embryonic Stem Cells via Small Molecule-Mediated Transient Alteration of Cell Structure. J Mater Chem B. 2014;2(46):8098-8105. PMID: 26005572.
Zhang Y, Yin Q, Yen J, Li J, Ying H, Wang H, Hua Y, Chaney EJ, Boppart SA, Cheng J. Non-invasive, real-time reporting drug release in vitro and in vivo. Chem Commun (Camb). 2015 Apr 25;51(32):6948-51. Epub 2015 Mar 23. PMID: 25798457.
Yen J, Zhang Y, Gabrielson NP, Yin L, Guan L, Chaudhury I, Lu H, Wang F, Cheng J. Cationic, helical polypeptide-based gene delivery for IMR-90 fibroblasts and human embryonic stem cells. Biomater Sci. 2013 Jul;1(7):719-727. PMID: 23997932. • Mali P, Ye Z, Chou BK, Yen J, Cheng L. An improved method for generating and identifying human induced pluripotent stem cells. Methods Mol Biol. 2010;636:191-205. PMID: 20336524.
Ohm JE, Mali P, Van Neste L, Berman DM, Liang L, Pandiyan K, Briggs KJ, Zhang W, Argani P, Simons B, Yu W, Matsui W, Van Criekinge W, Rassool FV, Zambidis E, Schuebel KE, Cope L, Yen J, Mohammad HP, Cheng L, Baylin SB. Cancerrelated epigenome changes associated with reprogramming to induced pluripotent stem cells. Cancer Res. 2010 Oct 1;70(19):7662-73. doi: 10.1158/0008-5472.CAN-10-1361. Epub 2010 Sep 14. PMID: 20841480.
Mali P, Chou BK, Yen J, Ye Z, Zou J, Dowey S, Brodsky RA, Ohm JE, Yu W, Baylin SB, Yusa K, Bradley A, Meyers DJ, Mukherjee C, Cole PA, Cheng L. Butyrate greatly enhances derivation of human induced pluripotent stem cells by promoting epigenetic remodeling and the expression of pluripotency-associated genes. Stem Cells. 2010 Apr;28(4):713- 20. PMID: 20201064.
Blelloch R, Venere M, Yen J, Ramalho-Santos M. Generation of induced pluripotent stem cells in the absence of drug selection. Cell Stem Cell. 2007 Sep 13;1(3):245-7. Epub 2007 Sep 6. PMID: 18371358.
Last update: August 2024
#Equal contribution
*Co-communicating senior authors