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Journal Articles

Adenine Base Editing Improves Erythropoiesis in Diamond-Blackfan Anemia Syndrome Patient-Derived Induced Pluripotent Stem Cells

Shruthi Suryaprakash, Lei Han, Garret Manquen, Varun Katta, Damian Krzyzanowski, Jayaram Prasad, Nikitha Nimmagada, Kalin Mayberry, Nana Liu, Yan Ju, Yu Yao, Kelsey Ray, Marcin Wlodarski, Shengdar Q Tsai, Jonathan S Yen, Mitchell J Weiss, Senthil Velan Bhoopalan
Journal: Blood
Blood (2024) 144 (Supplement 1): 4088.
https://doi.org/10.1182/blood-2024-209312
Published: 2024
Journal Articles

miRNA-144/451 Regulates Cell Surface TfR1 Expression in Normal and β-Thalassemic Erythroblasts

Georgios E. Christakopoulos, Alfonso G. Fernandez, Yu Yao, Rahul Telange, Dudley W. Michael, Tomas Ganz, Elizabeta Nemeth, Mitchell J Weiss
Journal: Blood
Blood (2024) 144 (Supplement 1): 2478.
https://doi.org/10.1182/blood-2024-210302
Published: 2024
Journal Articles

Iron Restriction Alleviates β-Thalassemia By Stimulating ULK1-Mediated Autophagy of Free α-Globin

Alfonso G. Fernandez, Georgios E. Christakopoulos, Dudley E. Michael, Razan B. A. Sweileh, Rahul Telange, Heather Sheppard, Yu Yao, Jingjing Zhang, Vania Manolova, Elizabeta Nemeth, Tomas Ganz, Mitchell J. Weiss
Journal: Blood
Blood (2023) 142 (Supplement 1): 558.
https://doi.org/10.1182/blood-2023-185008
Published: 2023
Journal Articles

FLT3 Testing and Clonal Evolution in Patients with Acute Myeloid Leukemia: A Retrospective Cohort Study in Taiwan

Xavier Cheng-Hong Tsai, Hwei-Fang Tien, Wen-Chien Chou, Prabhuram Krishnan, Yu-Yao Tai, Hsin-An Hou
Journal: Blood
Blood (2023) 142 (Supplement 1): 7422.
https://doi.org/10.1182/blood-2023-180617
Published: 2023
Journal Articles

Reduced Polycomb Repressor Complex 2 (PRC2) Activity and Increased TP53 Activity Mediate Hematopoietic Stem Cell Dysfunction in RPS19-Mutated Diamond-Blackfan Anemia

Senthil V Bhoopalan, Ruopeng Feng, Thiyagaraj Mayuranathan, Kalin Mayberry, Yoonjeong Jang, Jonathan S Yen, Yu Yao, Lance Palmer, Yong Cheng, Marcin W Wlodarski, Mitchell J. Weiss
Journal: Blood
Blood (2023) 142 (Supplement 1): 2732.
https://doi.org/10.1182/blood-2023-190069
Published: 2023
Journal Articles

Loss of miR-144/451 alleviates β-thalassemia by stimulating ULK1-mediated autophagy of free α-globin

Julia Keith, Georgios E. Christakopoulos, Alfonso G. Fernandez, Yu Yao, Jingjing Zhang, Kalin Mayberry, Rahul Telange, Razan B. A. Sweileh, Michael Dudley, Camilla Westbrook, Heather Sheppard, Mitchell J. Weiss, Christophe Lechauve
Journal: Blood
Blood (2023) 142 (10): 918–932.
https://doi.org/10.1182/blood.2022017265
Published: 2023
Includes: Supplemental data
Journal Articles

Increased Potency and Uniformity of Fetal Hemoglobin Induction from Base Editing Compared to Cas9 Nuclease

Thiyagaraj Mayuranathan, Gregory A. Newby, Ruopeng Feng, Yu Yao, Kalin Mayberry, Guolian Kang, Cicera Lazzarotto, Yichao Li, Rachel Levine, Erin Dempsey, Shaina N. Porter, Phillip A Doerfler, Jingjing Zhang, Yoonjeong Jang, Senthil Bhoopalan, Nikitha Nimmagadda, Akshay Sharma, John Tisdale, Shondra Miller, Yong Cheng, Shengdar Tsai, Mitchell J. Weiss, David R. Liu, Jonathan S Yen
Journal: Blood
Blood (2022) 140 (Supplement 1): 7784–7785.
https://doi.org/10.1182/blood-2022-162639
Published: 2022
Journal Articles

Germline Loss-of-Function Mutations in MDM4 Cause p53-Dependent Hematopoietic Cell Death in Patients with Variable Bone Marrow Failure Phenotypes

Richa Sharma, Senthil Bhoopalan, Lei Han, Yu Yao, Kalin Mayberry, Baranda S Hansen, Shondra Miller, Claudia Khurana, Miriam Erlacher, Marcin W Wlodarski
Journal: Blood
Blood (2022) 140 (Supplement 1): 2955–2956.
https://doi.org/10.1182/blood-2022-162614
Published: 2022
Journal Articles

Base Editing for Therapeutic Induction of Fetal Hemoglobin in β-Thalassemia

Georgios E. Christakopoulos, Thiyagaraj Mayuranathan, Alfonso G. Fernandez, Kalin Mayberry, Yu Yao, Rahul Telange, Michael Dudley, Rachel Levine, Erin Dempsey, David R. Liu, Jonathan S Yen, Mitchell J. Weiss
Journal: Blood
Blood (2022) 140 (Supplement 1): 10660–10661.
https://doi.org/10.1182/blood-2022-169391
Published: 2022
Journal Articles

Regulation of Fetal Hemoglobin Expression By the VHL-HIF1α Oxygen Sensing System

Ruopeng Feng, Thiyagaraj Mayuranathan, Peng Huang, Phillip A Doerfler, Yichao Li, Yu Yao, Jingjing Zhang, Lance E. Palmer, Peng Xu, Kalin Mayberry, Shaela Wright, Chunliang Li, Yong Cheng, Gerd A. Blobel, M. Celeste Simon, Mitchell J. Weiss
Journal: Blood
Blood (2021) 138 (Supplement 1): 574.
https://doi.org/10.1182/blood-2021-149901
Published: 2021
Journal Articles

A Novel RPS19 -Edited Hematopoietic Stem Cell Model of Diamond-Blackfan Anemia for Development of Lentiviral Vector Gene Therapy

Senthil Velan Bhoopalan, Jonathan Yen, Thiyagaraj Mayuranathan, Yu Yao, Kalin Mayberry, Sheng Zhou, Robert Throm, Marcin W. Wlodarski, Mitchell J. Weiss
Journal: Blood
Blood (2021) 138 (Supplement 1): 859.
https://doi.org/10.1182/blood-2021-147557
Published: 2021
Journal Articles

FBXO11-mediated proteolysis of BAHD1 relieves PRC2-dependent transcriptional repression in erythropoiesis

Peng Xu, Daniel C. Scott, Beisi Xu, Yu Yao, Ruopeng Feng, Li Cheng, Kalin Mayberry, Yong-Dong Wang, Wenjian Bi, Lance E. Palmer, Moeko T. King, Hong Wang, Yuxin Li, Yiping Fan, Arno F. Alpi, Chunliang Li, Junmin Peng, James Papizan, Shondra M. Pruett-Miller, Ria Spallek, Florian Bassermann, Yong Cheng, Brenda A. Schulman, Mitchell J. Weiss
Journal: Blood
Blood (2021) 137 (2): 155–167.
https://doi.org/10.1182/blood.2020007809
Published: 2021
Includes: Supplemental data
Journal Articles

Adenosine Base Editing of γ-Globin Promoters Induces Fetal Hemoglobin and Inhibit Erythroid Sickling

Thiyagaraj Mayuranathan, Jonathan S. Yen, PhD, Gregory A. Newby, PhD, Yu Yao, MD, Shaina N. Porter, PhD, Kaitly J. Woodard, BS, Jingjing Zhang, MS, Kalin Mayberry, BS, Akshay Sharma, MBBS, Shondra M. preutt-Miller, PhD, John F. Tisdale, MD, Shengdar Q. Tsai, PhD, David R. Liu, PhD, Mitchell J. Weiss, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 21–22.
https://doi.org/10.1182/blood-2020-141498
Published: 2020
Journal Articles

Base Editing Eliminates the Sickle Cell Mutation and Pathology in Hematopoietic Stem Cells Derived Erythroid Cells

Jonathan S Yen, PhD, Gregory A. Newby, PhD, Thiyagaraj Mayuranathan, Shaina N. Porter, PhD, Yu Yao, MD, Kaitly J. Woodard, BS, Kalin Mayberry, BS, Kelcee Everette, Jingjing Zhang, MS, Jordana M Henderson, MS, Anton P Mccaffrey, Jonathan Yen, John F. Tisdale, MD, Shengdar Q. Tsai, PhD, David R. Liu, PhD, Mitchell J. Weiss, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 13–14.
https://doi.org/10.1182/blood-2020-139016
Published: 2020
Journal Articles

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

Jean-Yves Metais, PhD, Phillip A Doerfler, PhD, Thiyagaraj Mayuranathan, Daniel E. Bauer, MD PhD, Stephanie Fowler, Matthew Hsieh, MD, Varun Katta, Sagar Keriwala, Cicera Lazzarotto, Kevin Luk, Michael Neel, Scott Perry, Shaina Porter, Byoung Y. Ryu, PhD, Akshay Sharma, MBBS, Devlin Shea, Samuel Peters, John F. Tisdale, MD, Scot A. Wolfe, PhD, Kaitly Jensen Woodard, BS, Naoya Uchida, MD, Yuxuan Wu, PhD, Yu Yao, MD, Jing Zeng, MD, Shondra M. Pruett-Miller, PhD, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2066.
https://doi.org/10.1182/blood-2019-131318
Published: 2019
Journal Articles

Regulation of gene expression by miR-144/451 during mouse erythropoiesis

Peng Xu, Lance E. Palmer, Christophe Lechauve, Guowei Zhao, Yu Yao, Jing Luan, Anastasios Vourekas, Haiyan Tan, Junmin Peng, John D. Schuetz, Zissimos Mourelatos, Gang Wu, Mitchell J. Weiss, Vikram R. Paralkar
Journal: Blood
Blood (2019) 133 (23): 2518–2528.
https://doi.org/10.1182/blood.2018854604
Published: 2019
Includes: Supplemental data
Journal Articles

The DNA Methylation Maintenance Protein UHRF1 Regulates Fetal Globin Expression Independent of HBG Promoter DNA Methylation

Ruopeng Feng, PhD, Phillip A Doerfler, PhD, Yu Yao, MD, Xing Tang, PhD, Yong-Dong Wang, PhD, Chunliang Li, PhD, David E. Root, PhD, Yong Cheng, PhD, Mitchell J. Weiss, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 410.
https://doi.org/10.1182/blood-2018-99-115589
Published: 2018
Journal Articles

FBXO11 Activates Erythroid Gene Transcription By Degrading Heterochromatin-Associated Protein BAHD1

Peng Xu, PhD, Daniel C. Scott, PhD, Xing Tang, PhD, Yu Yao, MD, Yong-Dong Wang, PhD, Wenjian Bi, PhD, Lance E. Palmer, PhD, Ruopeng Feng, PhD, Arno F. Alpi, PhD, Chunliang Li, PhD, Yong Cheng, PhD, Brenda A. Schulman, PhD, Mitchell J. Weiss, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 529.
https://doi.org/10.1182/blood-2018-99-117362
Published: 2018
Journal Articles

Nonspecific inhibition of erythropoiesis by short hairpin RNAs

Elizabeth A. Traxler, Christopher S. Thom, Yu Yao, Vikram Paralkar, Mitchell J. Weiss
Journal: Blood
Blood (2018) 131 (24): 2733–2736.
https://doi.org/10.1182/blood-2018-03-841304
Published: 2018
Journal Articles

Erythroid miRNA-144/451 Binds Many mRNAs but Regulates Only a Small Subset

Vikram R Paralkar, MD, Lance Palmer, PhD, Peng Xu, PhD, Christophe Lechauve, PhD, Guowei Zhao, PhD, Yu Yao, MD, Gang Wu, PhD, Jing Luan, Mitchell J. Weiss, MD PhD
Journal: Blood
Blood (2016) 128 (22): 1198.
https://doi.org/10.1182/blood.V128.22.1198.1198
Published: 2016