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Journal Articles

SAGES1: Clinical Translation of CRISPR Genome Editing Strategy to Induce Fetal Hemoglobin to Treat Sickle Cell Disease

Rachel M Levine, Erin A Dempsey, Nikitha Nimmagadda, Yoonjeong Jang, Kalin Mayberry, Jingjing Zhang, David Cullins, Catherine Willis, Alicia Powers, Young-In Kim-Hoehammer, Jie Lin, Fei Zhang, Neha Das Gupta, Will Lewis, Varun Katta, Yichao Li, Timothy Lockey, Yong Cheng, Patti Lehigh, Frank Fazio, Alexis Kristine Leonard, Akshay Sharma, Sheng Zhou, Shengdar Q Tsai, Mitchell J Weiss, Jonathan S Yen
Journal: Blood
Blood (2024) 144 (Supplement 1): 146.
https://doi.org/10.1182/blood-2024-211182
Published: 2024
Journal Articles

CD123-CAR T Cells Manufactured in the Presence of Dasatinib Induce High Grade CRS and/or IEC-HS without Improving Efficacy in Pediatric Patients with Recurrent/Refractory Leukemia

Swati Naik, Madden Renee, Aimee C. Talleur, Rebecca Epperly, Timothy Lockey, Jennyfer Bran, Liqing Tian, Ying Li, Dinesh Keerthi, Alexandra Boyd, Amanda Lipsitt, Jeffrey E. Rubnitz, Jeffery M Klco, Barry Shulkin, Sarah Schell, Polly Adams, Jeoungeun John Park, Janice Riberdy, Na Shang, Jaquelyn T. Zoine, Raghuvaran Shanmugam, Chris Nevitt, Unmesha Thanekar, Jennifer Wallace, Elaine Harstead, Scott Perry, Catherine Willis, Jean-Yves Metais, Deanna M. Langfitt, Sheng Zhou, Salem M. Akel, Caitlin Zebley, Jeremy Chase Crawford, Benjamin Youngblood, Paul G. Thomas, Frank Fazio, Brandon M Triplett, Stephen Gottschalk, Mireya Paulina Velasquez
Journal: Blood
Blood (2024) 144 (Supplement 1): 2076.
https://doi.org/10.1182/blood-2024-208795
Published: 2024
Journal Articles

Safety and Anti-Leukemic Activity of CD123-CAR T Cells in Pediatric Patients with AML: Preliminary Results from a Phase 1 Trial

Swati Naik, Renee M. Madden, Amanda Lipsitt, Timothy Lockey, Jennyfer Bran, Jeffrey E. Rubnitz, Jeffery Klco, Barry Shulkin, Sagar L. Patil, Sarah Schell, Jeoungeun John Park, Janice Riberdy, Na Shang, Jaquelyn T. Zoine, Jennifer Wallace, Elaine Harstead, Catherine Willis, Jean-Yves Metais, Deanna M. Langfitt, Sheng Zhou, Salem M. Akel, Michael M. Meagher, Brandon M. Triplett, Stephen Gottschalk, Mireya Paulina Velasquez
Journal: Blood
Blood (2022) 140 (Supplement 1): 4584–4585.
https://doi.org/10.1182/blood-2022-170201
Published: 2022
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

Allogeneic Hematopoietic Cell Transplantation Is Critical to Maintain Remissions after CD19-CAR T-Cell Therapy for Pediatric ALL: A Single Center Experience

Aimee C Talleur, MD, Renee M. Madden, MS, MD, Amr Qudeimat, MD, Ewelina Mamcarz, MD, Akshay Sharma, MBBS, Ashok Srinivasan, MD, Ali Y Suliman, MD, Mireya Paulina Velasquez, MD, Timothy Lockey, PhD, Alisha Gaboriault, Seth E. Karol, MD, Hiroto Inaba, MD PhD, Ying Li, MD PhD, Pam Young, Guolian Kang, PhD, Cheng Cheng, PhD, Wenting Zheng, PhD, Sheng Zhou, PhD, Salem M. Akel, PhD, Terrence L Geiger, MD PhD, Michael M Meagher, PhD, Ching-Hon Pui, MD, Brandon M Triplett, MD, Stephen Gottschalk, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 39–40.
https://doi.org/10.1182/blood-2020-138528
Published: 2020
Journal Articles

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study

Ewelina Mamcarz, MD, Sheng Zhou, PhD, Timothy Lockey, PhD, Shannon Boi, PhD, Yan Koon-Kiu, PhD, Shane Cross, Pharm.D., Guolian Kang, PhD, Zhijun Ma, MD, Jose Marcos Condori, PhD, Jolanta Dowdy, M.S., Jean-Yves Metais, PhD, Deanna Langfitt, PhD, Brandon Triplett, MD, Chen Li, M.S., Xiwen Zhao, MSPH, Gabriela Maron, MD, Sneha Suresh, MD, Juan Carlos Aldave Becerra, MD, Joseph Church, MD, Elif Dokmeci, MD, James T Love, MD, PhD, Hedi van der Watt, MD, Ana C da Matta Ain, MD, Christa Krupski, DO, MPH, Jiyang Yu, PhD, Lance E. Palmer, PhD, William Janssen, PhD, Suk See De Ravin, MD, PhD, Mitchell J. Weiss, MD, PhD, Benjamin Youngblood, PhD, Janel R Long-Boyle, PharmD, PhD, Michael M Meagher, PhD, Harry L. Malech, MD, Jennifer Puck, MD, Morton J Cowan, MD, Stephen Gottschalk, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2058.
https://doi.org/10.1182/blood-2019-126746
Published: 2019
Journal Articles

Interim Results from a Phase I/II Clinical Gene Therapy Study for Newly Diagnosed Infants with X-Linked Severe Combined Immunodeficiency Using a Safety-Modified Lentiviral Vector and Targeted Reduced Exposure to Busulfan

Ewelina Mamcarz, MD, Sheng Zhou, PhD, Timothy Lockey, PhD, Hossam Abdelsamed, PhD, Shane Cross, Pharm D, Zhijun Ma, MD, Jola Dowdy, Brandon Triplett, MD, Mitchell J. Weiss, MD PhD, Byoung Y. Ryu, PhD, Elif Dokmeci, MD, Juan Carlos Aldave, MD, Suk See De Ravin, MD PhD, Benjamin Youngblood, PhD, Janel R. Long-Boyle, PharmD, PhD, Michael M Meagher, PhD, Harry L Malech, MD, Jennifer Puck, MD, Morton Jerome Cowan, MD, Brian Sorrentino, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 523.
https://doi.org/10.1182/blood.V130.Suppl_1.523.523
Published: 2017
Journal Articles

Downregulation of Prdm16 mRNA is a specific antileukemic mechanism during HOXB4-mediated HSC expansion in vivo

Hui Yu, Geoffrey Neale, Hui Zhang, Han M. Lee, Zhijun Ma, Sheng Zhou, Bernard G. Forget, Brian P. Sorrentino
Journal: Blood
Blood (2014) 124 (11): 1737–1747.
https://doi.org/10.1182/blood-2013-10-534735
Published: 2014
Includes: Supplemental data
Journal Articles

A Role For a Multi-Drug Resistance Protein (MRP4/ABCC4) In Pediatric Acute Myeloid Leukemia (AML)

Shang Li Lian, PhD, Yao WANG, Sheng Zhou, Mugdha Sukthankar, Geoffrey A. Neale, PhD, James R Downing, MD, John D. Schuetz, PhD
Journal: Blood
Blood (2013) 122 (21): 2518.
https://doi.org/10.1182/blood.V122.21.2518.2518
Published: 2013
Journal Articles

N-Myc Is Overexpressed In Both Murine and Human Early T-Cell Precursor Leukemia and Is Sufficient To Initiate this Leukemia In Multipotent Primitive Arf -/- thymocytes

Louise M. Treanor, PhD, Sheng Zhou, PhD, Yu Fukuda, PhD, Nandakumar Satish, David Finkelstein, PhD, John D. Schuetz, PhD, Charles G. Mullighan, MBBS, MSc, MD, FRACP, FRCPA, Brian P. Sorrentino, MD
Journal: Blood
Blood (2013) 122 (21): 348.
https://doi.org/10.1182/blood.V122.21.348.348
Published: 2013
Journal Articles

Gene Expression Profiling of Murine HOXB4-Transduced HSCs Shows Multiple Counter-Regulatory Signals That May Control HSC Pool Size

Hui Yu, Sheng Zhou, PhD, Geoffrey A. Neale, PhD, Brian P. Sorrentino, MD
Journal: Blood
Blood (2011) 118 (21): 2361.
https://doi.org/10.1182/blood.V118.21.2361.2361
Published: 2011
Journal Articles

Marking of Embryonic Precursors of Adult Hematopoietic Stem Cells At Gestational Day E7.5-E8.5 Using An Abcg2-CreER T2 Lineage Tracing Mouse Model

Sheng Zhou, PhD, Soghra Fatima, PhD, Brian P. Sorrentino, MD
Journal: Blood
Blood (2011) 118 (21): 1287.
https://doi.org/10.1182/blood.V118.21.1287.1287
Published: 2011
Journal Articles

Lmo2 Overexpression and Arf Loss Induce Myeloid Differentiation in Primitive Thymocytes

Louise M. Treanor, PhD, Sheng Zhou, PhD, Taihe Lu, PhD, Charles G Mullighan, MBBS, MD, FRACP, FRCPA, MSc, Brian P. Sorrentino, MD
Journal: Blood
Blood (2011) 118 (21): 47.
https://doi.org/10.1182/blood.V118.21.47.47
Published: 2011
Journal Articles

Functional interactions between Lmo2, the Arf tumor suppressor, and Notch1 in murine T-cell malignancies

Louise M. Treanor, Emmanuel J. Volanakis, Sheng Zhou, Taihe Lu, Charles J. Sherr, Brian P. Sorrentino
Journal: Blood
Blood (2011) 117 (20): 5453–5462.
https://doi.org/10.1182/blood-2010-09-309831
Published: 2011
Includes: Supplemental data
Journal Articles

Loss of p19 Arf Increases the Self-Renewal Capacity of Immature Thymocytes That Over-Express LMO2

Louise M. Treanor, PhD, Sheng Zhou, PhD, Taihe Lu, Brian P. Sorrentino, MD
Journal: Blood
Blood (2010) 116 (21): 4198.
https://doi.org/10.1182/blood.V116.21.4198.4198
Published: 2010
Journal Articles

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

Sheng Zhou, Disha Mody, Suk See DeRavin, Julia Hauer, Taihe Lu, Zhijun Ma, Salima Hacein-Bey Abina, John T. Gray, Michael R. Greene, Marina Cavazzana-Calvo, Harry L. Malech, Brian P. Sorrentino
Journal: Blood
Blood (2010) 116 (6): 900–908.
https://doi.org/10.1182/blood-2009-10-250209
Published: 2010
Includes: Supplemental data
Journal Articles

Hemgn is a direct transcriptional target of HOXB4 and induces expansion of murine myeloid progenitor cells

Jie Jiang, Hui Yu, Yan Shou, Geoffrey Neale, Sheng Zhou, Taihe Lu, Brian P. Sorrentino
Journal: Blood
Blood (2010) 116 (5): 711–719.
https://doi.org/10.1182/blood-2009-07-235341
Published: 2010
Includes: Supplemental data
Journal Articles

Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection

Robert E. Throm, Annastasia A. Ouma, Sheng Zhou, Anantharaman Chandrasekaran, Timothy Lockey, Michael Greene, Suk See De Ravin, Morvarid Moayeri, Harry L. Malech, Brian P. Sorrentino, John T. Gray
Journal: Blood
Blood (2009) 113 (21): 5104–5110.
https://doi.org/10.1182/blood-2008-11-191049
Published: 2009
Journal Articles

Abcg2 Expression Marks Neural Stem Cells in the Murine Cerebellum.

Sheng Zhou, PhD, Soghra Fatima, Ph.D, Brian P. Sorrentino, M.D.
Journal: Blood
Blood (2006) 108 (11): 1674.
https://doi.org/10.1182/blood.V108.11.1674.1674
Published: 2006
Journal Articles

Expression of Abcg2 in Murine Skeletal Muscle Cells Identifies Two Populations with Different Myogenic Potential.

Mehrdad Tadjali, PhD, Sheng Zhou, Ph.D, Brian P. Sorrentino, M.D.
Journal: Blood
Blood (2006) 108 (11): 1669.
https://doi.org/10.1182/blood.V108.11.1669.1669
Published: 2006