Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells

Authors: Zeng, Jing ^{1, 2, 3, 4}, Nguyen, My Anh ^{1, 2, 3, 4}, Liu, Pengpeng ⁵, da Silva, Lucas Ferreira ^{1, 2, 3, 4, 6, 7}, Levesque, Sébastien ^{1, 2, 3, 4}, Lin, Linda Y. ^{1, 2, 3, 4}, Justus, David G. ^{7, 8}, Petri, Karl ^{6, 7}, Clement, Kendell ^{6, 7}, Porter, Shaina N. ⁹, Verma, Archana ^{1, 2, 3, 4}, Neri, Nola R. ^{1, 2, 3, 4}, Rosanwo, Tolulope ^{1, 2, 3, 4}, Ciuculescu, Marioara-Felicia ¹⁰, Abriss, Daniela ¹⁰, Mintzer, Esther ⁵, Maitland, Stacy A. ⁵, Demirci, Selami ¹¹, Cha, Hye Ji ^{1, 2, 3, 4, 13}, Orkin, Stuart H. ^{1, 2, 3, 4}, Tisdale, John F. ¹¹, Williams, David A. ^{1, 2, 3, 4}, Zhu, Lihua Julie ¹², Pruett-Miller, Shondra M. ⁹, Pinello, Luca ^{6, 7}, Joung, J. Keith ^{6, 7}, Pattanayak, Vikram ^{6, 7}, Manis, John P. ^{1, 2, 3, 4}, Armant, Myriam ¹⁰, Pellin, Danilo ^{1, 2, 3, 4}, Brendel, Christian ^{1, 2, 3, 4}, Wolfe, Scot A. ⁵, Bauer, Daniel E. ^{1, 2, 3, 4, 14, ∗}

Source: In Cell Stem Cell 6 February 2025 32(2):191-208

Redirecting E3 ubiquitin ligases for targeted protein degradation with heterologous recognition domains

Authors: Yang, Huan ^{1, 2}, Zheng, Ge ^{1, 2}, Li, Grace Y. ¹, Alshaye, Alia ¹, Orkin, Stuart H. ^{1, 2, 3, ∗}

Source: In Journal of Biological Chemistry January 2025 301(1)

Structural insights into the DNA-binding mechanism of BCL11A: The integral role of ZnF6

Authors: Viennet, Thibault, Yin, Maolu, Jayaraj, Abhilash, Kim, Woojin, Sun, Zhen-Yu J., Fujiwara, Yuko, Zhang, Kevin, Seruggia, Davide, Seo, Hyuk-Soo, Dhe-Paganon, Sirano, Orkin, Stuart H., Arthanari, Haribabu

Source: In Structure 5 December 2024 32(12):2276-2286

Mechanistic and kinetic insights into transcription factor biology via acute protein depletion

Authors: Mehta, Stuti, Buyanbat, Altantsetseg, Kai, Yan, Orkin, Stuart H.

Source: In Experimental Hematology September 2024 137

In vivo CRISPR/Cas9 screening identifies Pbrm1 as a regulator of myeloid leukemia development in mice

Authors: Li, Bin E., Li, Grace Y., Cai, Wenqing, Zhu, Qian, Seruggia, Davide, Fujiwara, Yuko, Vakoc, Christopher R., Orkin, Stuart H.

Source: In Blood Advances 26 September 2023 7(18):5281-5293

Fetal Hemoglobin Regulation in Beta-Thalassemia

Authors: Lu, Henry Y., Orkin, Stuart H., Sankaran, Vijay G.

Source: In Hematology/Oncology Clinics of North America April 2023 37(2):301-312

Congenital anemia reveals distinct targeting mechanisms for master transcription factor GATA1

Authors: Ludwig, Leif S., Lareau, Caleb A., Bao, Erik L., Liu, Nan, Utsugisawa, Taiju, Tseng, Alex M., Myers, Samuel A., Verboon, Jeffrey M., Ulirsch, Jacob C., Luo, Wendy, Muus, Christoph, Fiorini, Claudia, Olive, Meagan E., Vockley, Christopher M., Munschauer, Mathias, Hunter, Abigail, Ogura, Hiromi, Yamamoto, Toshiyuki, Inada, Hiroko, Nakagawa, Shinichiro, Ohzono, Shuichi, Subramanian, Vidya, Chiarle, Roberto, Glader, Bertil, Carr, Steven A., Aryee, Martin J., Kundaje, Anshul, Orkin, Stuart H., Regev, Aviv, McCavit, Timothy L., Kanno, Hitoshi, Sankaran, Vijay G.

Source: Blood. 139(16):2534-2546

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100183251461.0x000001&indx=1&recIds=ETOCvdc_100183251461.0x000001

CD49b identifies functionally and epigenetically distinct subsets of lineage-biased hematopoietic stem cells

Authors: Somuncular, Ece, Hauenstein, Julia, Khalkar, Prajakta, Johansson, Anne-Sofie, Dumral, Özge, Frengen, Nicolai S., Gustafsson, Charlotte, Mocci, Giuseppe, Su, Tsu-Yi, Brouwer, Hugo, Trautmann, Christine L., Vanlandewijck, Michael, Orkin, Stuart H., Månsson, Robert, Luc, Sidinh

Source: Stem cell reports. 17(7):1546-1560

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100169234004.0x000001&indx=1&recIds=ETOCvdc_100169234004.0x000001

Temporal resolution of gene derepression and proteome changes upon PROTAC-mediated degradation of BCL11A protein in erythroid cells

Authors: Mehta, Stuti, Buyanbat, Altantsetseg, Kai, Yan, Karayel, Ozge, Goldman, Seth Raphael, Seruggia, Davide, Zhang, Kevin, Fujiwara, Yuko, Donovan, Katherine A., Zhu, Qian, Yang, Huan, Nabet, Behnam, Gray, Nathanael S., Mann, Matthias, Fischer, Eric S., Adelman, Karen, Orkin, Stuart H.

Source: In Cell Chemical Biology 18 August 2022 29(8):1273-1287

Hypoxic, glycolytic metabolism is a vulnerability of B-acute lymphoblastic leukemia-initiating cells

Authors: Morris, Vivian, Wang, Dahai, Li, Zhiheng, Marion, William, Hughes, Travis, Sousa, Patricia, Harada, Taku, Sui, Shannan Ho, Naumenko, Sergey, Kalfon, Jérémie, Sensharma, Prerana, Falchetti, Marcelo, Vinicius da Silva, Renan, Candelli, Tito, Schneider, Pauline, Margaritis, Thanasis, Holstege, Frank C.P., Pikman, Yana, Harris, Marian, Stam, Ronald W., Orkin, Stuart H., Koehler, Angela N., Shalek, Alex K., North, Trista E., Pimkin, Maxim, Daley, George Q., Lummertz da Rocha, Edroaldo, Rowe, R. Grant

Source: In Cell Reports 26 April 2022 39(4)

Developmental maturation of the hematopoietic system controlled by a Lin28b-let-7-Cbx2 axis

Authors: Wang, Dahai, Tanaka-Yano, Mayuri, Meader, Eleanor, Kinney, Melissa A., Morris, Vivian, Lummertz da Rocha, Edroaldo, Liu, Nan, Liu, Tianxin, Zhu, Qian, Orkin, Stuart H., North, Trista E., Daley, George Q., Rowe, R. Grant

Source: In Cell Reports 5 April 2022 39(1)

Enhancer dependence of cell-type–specific gene expression increases with developmental age

Authors: Cai, Wenqing, Huang, Jialiang, Zhu, Qian, Li, Bin E., Seruggia, Davide, Zhou, Pingzhu, Nguyen, Minh, Fujiwara, Yuko, Xie, Huafeng, Yang, Zhenggang, Hong, Danni, Ren, Pengfei, Xu, Jian, Pu, William T., Yuan, Guo-Cheng, Orkin, Stuart H.

Source: Proceedings of the National Academy of Sciences of the United States of America, 2020 Sep 01. 117(35), 21450-21458.

Access URL: https://www.jstor.org/stable/26968818

Dietary suppression of MHC class II expression in intestinal epithelial cells enhances intestinal tumorigenesis

Authors: Beyaz, Semir, Chung, Charlie, Mou, Haiwei, Bauer-Rowe, Khristian E., Xifaras, Michael E., Ergin, Ilgin, Dohnalova, Lenka, Biton, Moshe, Shekhar, Karthik, Eskiocak, Onur, Papciak, Katherine, Ozler, Kadir, Almeqdadi, Mohammad, Yueh, Brian, Fein, Miriam, Annamalai, Damodaran, Valle-Encinas, Eider, Erdemir, Aysegul, Dogum, Karoline, Shah, Vyom, Alici-Garipcan, Aybuke, Meyer, Hannah V., Özata, Deniz M., Elinav, Eran, Kucukural, Alper, Kumar, Pawan, McAleer, Jeremy P., Fox, James G., Thaiss, Christoph A., Regev, Aviv, Roper, Jatin, Orkin, Stuart H., Yilmaz, Ömer H.

Source: In Cell Stem Cell 4 November 2021 28(11):1922-1935

Molecular Medicine: Found in Translation

Authors: Orkin, Stuart H.

Source: In Med 12 February 2021 2(2):122-136

A tetramer of BCL11A is required for stable protein production and fetal hemoglobin silencing.

Authors: Ge Zheng¹, Maolu Yin¹, Mehta, Stuti¹, I-Te Chu¹, Wang, Stacy², AlShaye, Alia¹, Drainville, Kirstin¹, Buyanbat, Altantsetseg¹, Bienfait, Frédérique¹, Tenglin, Karin¹, Qian Zhu², Orkin, Stuart H.^1,3, stuart_orkin@dfci.harvard.edu

Source: Science; 11/29/2024, Vol. 386 Issue 6725, p1010-1018, 9p, 6 Diagrams

ARID4B is critical for mouse embryonic stem cell differentiation towards mesoderm and endoderm, linking epigenetics to pluripotency exit

Authors: Terzi Cizmecioglu, Nihal ^*, Huang, Jialiang, Keskin, Ezgi G., Wang, Xiaofeng, Esen, Idil, Chen, Fei, Orkin, Stuart H. ^*

Source: In Journal of Biological Chemistry 18 December 2020 295(51):17738-17751

A saturating mutagenesis CRISPR-Cas9–mediated functional genomic screen identifies cis- and trans-regulatory elements of Oct4 in murine ESCs

Authors: Canver, Matthew C., Tripathi, Pratibha, Bullen, Michael J., Olshansky, Moshe, Kumar, Yogesh, Wong, Lee H., Turner, Stephen J., Lessard, Samuel, Pinello, Luca, Orkin, Stuart H. ^*, Das, Partha Pratim ^*

Source: In Journal of Biological Chemistry 20 November 2020 295(47):15797-15809

An Engineered CRISPR-Cas9 Mouse Line for Simultaneous Readout of Lineage Histories and Gene Expression Profiles in Single Cells

Authors: Bowling, Sarah, Sritharan, Duluxan, Osorio, Fernando G., Nguyen, Maximilian, Cheung, Priscilla, Rodriguez-Fraticelli, Alejo, Patel, Sachin, Yuan, Wei-Chien, Fujiwara, Yuko, Li, Bin E., Orkin, Stuart H., Hormoz, Sahand, Camargo, Fernando D.

Source: In Cell 11 June 2020 181(6):1410-1422

TAF5L and TAF6L Maintain Self-Renewal of Embryonic Stem Cells via the MYC Regulatory Network

Authors: Seruggia, Davide, Oti, Martin, Tripathi, Pratibha, Canver, Matthew C., LeBlanc, Lucy, Di Giammartino, Dafne C., Bullen, Michael J., Nefzger, Christian M., Sun, Yu Bo Yang, Farouni, Rick, Polo, Jose M., Pinello, Luca, Apostolou, Effie, Kim, Jonghwan, Orkin, Stuart H., Das, Partha Pratim

Source: In Molecular Cell 20 June 2019 74(6):1148-1163

Extensive Recovery of Embryonic Enhancer and Gene Memory Stored in Hypomethylated Enhancer DNA

Authors: Jadhav, Unmesh, Cavazza, Alessia, Banerjee, Kushal K., Xie, Huafeng, O’Neill, Nicholas K., Saenz-Vash, Veronica, Herbert, Zachary, Madha, Shariq, Orkin, Stuart H., Zhai, Huili, Shivdasani, Ramesh A.

Source: In Molecular Cell 2 May 2019 74(3):542-554