Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells

Authors: Chu, Simon N. ^{1, 2, 3}, Soupene, Eric ⁴, Sharma, Devesh ^{1, 2}, Sinha, Roshani ^{1, 2}, McCreary, Travis ^{1, 2}, Hernandez, Britney ⁵, Shen, Huifeng ⁵, Wienert, Beeke ⁶, Bowman, Chance ^{1, 2, 7}, Yin, Han ^{1, 3}, Lesch, Benjamin J. ^{1, 2, 7}, Jia, Kun ^{1, 2}, Romero, Kathleen A. ⁵, Kostamo, Zachary ⁵, Zhang, Yankai ⁵, Tran, Tammy ^{1, 2}, Cordero, Marco ^{1, 2}, Homma, Shota ⁸, Hampton, Jessica P. ⁹, Gardner, James M. ^{1, 3}, Conklin, Bruce R. ^{6, 10}, MacKenzie, Tippi C. ^{1, 2}, Sheehan, Vivien A. ⁵, Porteus, Matthew H. ⁹, Cromer, M. Kyle ^{1, 2, 7, 11, ∗}

Source: In Cell Reports 28 January 2025 44(1)

A differentiated β-globin gene replacement strategy uses heterologous introns to restore physiological expression

Authors: Wallace, Kirby A. ^{1, 2}, Gerstenberg, Trevor L. ¹, Ennis, Craig L. ¹, Perez-Bermejo, Juan A. ¹, Partridge, James R. ¹, Bandoro, Christopher ¹, Matern, William M. ¹, Andreoletti, Gaia ¹, Krassovsky, Kristina ¹, Kabir, Shaheen ¹, Lalisan, Cassandra D. ¹, Churi, Aishwarya R. ¹, Chew, Glen M. ¹, Corbo, Lana ¹, Vincelette, Jon E. ¹, Klasson, Timothy D. ¹, Silva, Brian J. ¹, Strukov, Yuri G. ¹, Quejarro, B. Joy ¹, Hill, Kaisle A. ¹, Treusch, Sebastian ¹, Grogan, Jane L. ¹, Dever, Daniel P. ¹, Porteus, Matthew H. ^{2, ∗}, Wienert, Beeke ^{1, ∗∗}

Source: In Molecular Therapy February 2025

Unbiased detection of CRISPR off-targets in vivo using DISCOVER-Seq

Authors: Wienert, Beeke, Wyman, Stacia K., Richardson, Christopher D., Yeh, Charles D., Akcakaya, Pinar, Porritt, Michelle J., Morlock, Michaela, Vu, Jonathan T., Kazane, Katelynn R., Watry, Hannah L., Judge, Luke M., Conklin, Bruce R., Maresca, Marcello, Corn, Jacob E.

Source: Science, 2019 Apr . 364(6437), 286-289.

Access URL: https://www.jstor.org/stable/26649347

Wake-up Sleepy Gene: Reactivating Fetal Globin for β-Hemoglobinopathies

Authors: Wienert, Beeke, Martyn, Gabriella E., Funnell, Alister P.W., Quinlan, Kate G.R., Crossley, Merlin

Source: In Trends in Genetics December 2018 34(12):927-940

A natural regulatory mutation in the proximal promoter elevates fetal globin expression by creating a de novo GATA1 site

Authors: Martyn, Gabriella E., Wienert, Beeke, Kurita, Ryo, Nakamura, Yukio, Quinlan, Kate G. R., Crossley, Merlin

Source: Blood. 133(8):852-856

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN619108941&indx=1&recIds=ETOCRN619108941

METTL21B Is a Novel Human Lysine Methyltransferase of Translation Elongation Factor 1A: Discovery by CRISPR/Cas9 Knockout

Authors: Hamey, Joshua J. ^‡, Wienert, Beeke ^‡, Quinlan, Kate G.R. ^‡, Wilkins, Marc R. ^{‡, §}

Source: In Molecular & Cellular Proteomics December 2017 16(12):2229-2242

KLF1 directly activates expression of the novel fetal globin repressor ZBTB7A/LRF in erythroid cells

Authors: Norton, Laura J., Funnell, Alister P.W., Burdach, Jon, Wienert, Beeke, Kurita, Ryo, Nakamura, Yukio, Philipsen, Sjaak, Pearson, Richard C.M., Quinlan, Kate G.R., Crossley, Merlin ^*

Source: In Blood Advances 25 April 2017 1(11):685-692

Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding

Authors: Martyn, Gabriella, Wienert, Beeke, Yang, Lu, Shah, Manan, Norton, Laura, Burdach, Jon, Kurita, Ryo, Nakamura, Yukio, Pearson, Richard, Funnell, Alister, Quinlan, Kate, Crossley, Merlin

Source: Nature genetics. 50(4):498-503

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100129200011.0x000001&indx=1&recIds=ETOCvdc_100129200011.0x000001

Phosphorylation of Krüppel-like Factor 3 (KLF3/BKLF) and C-terminal Binding Protein 2 (CtBP2) by Homeodomain-interacting Protein Kinase 2 (HIPK2) Modulates KLF3 DNA Binding and Activity

Authors: Dewi, Vitri, Kwok, Alister, Lee, Stella, Lee, Ming Min, Tan, Yee Mun, Nicholas, Hannah R., Isono, Kyo-ichi, Wienert, Beeke, Mak, Ka Sin, Knights, Alexander J., Quinlan, Kate G.R., Cordwell, Stuart J., Funnell, Alister P.W., Pearson, Richard C.M., Crossley, Merlin

Source: In Journal of Biological Chemistry 27 March 2015 290(13):8591-8605

KLF1 drives the expression of fetal hemoglobin in British HPFH

Authors: Wienert, Beeke, Martyn, Gabriella E., Kurita, Ryo, Nakamura, Yukio, Quinlan, Kate G. R., Crossley, Merlin

Source: Blood. 130(6):803-807

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN612365603&indx=1&recIds=ETOCRN612365603

Timed inhibition of CDC7 increases CRISPR-Cas9 mediated templated repair

Authors: Wienert, Beeke, Nguyen, David N., Guenther, Alexis, Feng, Sharon J., Locke, Melissa N., Wyman, Stacia K., Shin, Jiyung, Kazane, Katelynn R., Gregory, Georgia L., Carter, Matthew A. M., Wright, Francis, Conklin, Bruce R., Marson, Alex, Richardson, Chris D., Corn, Jacob E.

Source: Nature communications. 11(1)

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100174614703.0x000001&indx=1&recIds=ETOCvdc_100174614703.0x000001

Structural basis of simultaneous recruitment of the transcriptional regulators LMO2 and FOG1/ZFPM1 by the transcription factor GATA1

Authors: Wilkinson-White, Lorna, Gamsjaeger, Roland, Dastmalchi, Siavoush, Wienert, Beeke, Stokes, Philippa H., Crossley, Merlin, Mackay, Joel P., Matthews, Jacqueline M.

Source: Proceedings of the National Academy of Sciences of the United States of America, 2011 Aug . 108(35), 14443-14448.

Access URL: https://www.jstor.org/stable/27979311

In vitro–transcribed guide RNAs trigger an innate immune response via the RIG-I pathway.

Authors: Wienert, Beeke^1,2, Shin, Jiyung^1,2, Zelin, Elena^1,2, Pestal, Kathleen², Corn, Jacob E.^1,2 jcorn@berkeley.edu

Source: PLoS Biology. 7/16/2018, Vol. 16 Issue 7, p1-18. 18p.

Subject Terms: *CRISPRS, *NUCLEIC acid isolation methods, *IMMUNE response, *IN vitro studies, *BIOCHEMISTRY

Dual α-Globin and Truncated EPO Receptor Knockin Restores Hemoglobin Production in α-Thalassemia-Derived Hematopoietic Stem and Progenitor Cells

Authors: Chu, Simon N, Soupene, Eric, Wienert, Beeke, Yin, Han, Sharma, Devesh, Jia, Kun, Homma, Shota, Hampton, Jessica P, Conklin, Bruce, MacKenzie, Tippi C., Porteus, Matthew, Cromer, Kyle

Source: In Blood 2 November 2023 142 Supplement 1:485-485

Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin.

Authors: Ravi, Nithin Sam^1,2, Wienert, Beeke^3,4,5, Wyman, Stacia K.³, Bell, Henry William⁵, George, Anila^1,6, Mahalingam, Gokulnath¹, Vu, Jonathan T.³, Prasad, Kirti^1,7, Bandlamudi, Bhanu Prasad¹, Devaraju, Nivedhitha^1,7, Rajendiran, Vignesh^1,6, Syedbasha, Nazar¹, Pai, Aswin Anand^6,8, Yukio Nakamura⁹, Kurita, Ryo¹⁰, Narayanasamy, Muthuraman^1,11, Balasubramanian, Poonkuzhali^6,8, Thangavel, Saravanabhavan¹, Marepally, Srujan¹, Velayudhan, Shaji R.^1,6,8

Source: eLife. 2/23/2022, p1-32. 32p.

Subject Terms: *FETAL hemoglobin, *HEMATOPOIETIC stem cells, *CRISPRS, *GLOBIN, *BINDING sites

Development of a β-Globin Gene Replacement Strategy As a β-Thalassemia Therapy

Authors: Wallace, Kirby ^*, Wienert, Beeke ^*, Bandoro, Christopher ^*, Partridge, James ^*, Ennis, Craig L. ^*, Lalisan, Casey D. ^*, Krassovsky, Kristina ^*, Andreoletti, Gaia ^*, Sharma, Rajiv ^*, Churi, Aishwarya ^*, Matern, William M. ^*, Treusch, Sebastian ^*, Grogan, Jane ^*, Dever, Daniel P. ^*

Source: In Blood 15 November 2022 140 Supplement 1:12985-12986

CRISPR off-target detection with DISCOVER-seq.

Authors: Wienert, Beeke, Wyman, Stacia K., Yeh, Charles D., Conklin, Bruce R., Corn, Jacob E.

Source: Nature Protocols; May2020, Vol. 15 Issue 5, p1775-1799, 25p

Investigating Zeta Globin Gene Expression to Develop a Potential Therapy for Alpha Thalassemia Major

Authors: Gregory, Georgia L. ^*, Wienert, Beeke ^*, Schwab, Marisa ^*, Lianoglou, Billie Rachael ^*, Hollis, Roger P. ^*, Kohn, Donald B., Conklin, Bruce R. ^*, MacKenzie, Tippi C.

Source: In Blood 5 November 2020 136 Supplement 1:3-4

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A natural regulatory mutation in the proximal promoter elevates fetal globinexpression by creating a de novo GATA1 site

Authors: Martyn, Gabriella E., Wienert, Beeke, Kurita, Ryo, Nakamura, Yukio, Quinlan, Kate G.R., Crossley, Merlin

Source: Blood; February 2019, Vol. 133 Issue: 8 p852-856, 5p