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Michael M Meagher
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Journal Articles

GO-8: Stable Expression of Factor VIII over 5 Years Following Adeno-Associated Gene Transfer in Subjects with Hemophilia a Using a Novel Human Factor VIII Variant

Pratima Chowdary, Ulrike M. Reiss, Edward G. D. Tuddenham, Paul Batty, Jenny H. McIntosh, Vlad Calin Radulescu, Eugenia Chang, Michael A. Laffan, Anne Riddell, Joanna C. M. Calvert, Arnulfo Pie, Rita Peralta, Upuli Pabakumari Dissanayake, Kirollos Kamel, Bharath Ram Sreedhar, Junfang Zhou, Guolian Kang, Yunus Olufadi, Michael M Meagher, Christopher L Morton, Alison J. Evans, Andrew M. Davidoff, Amit C. Nathwani
Journal: Blood
Blood (2023) 142 (Supplement 1): 3624.
https://doi.org/10.1182/blood-2023-180803
Published: 2023
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

CD19-CAR T Cells Develop Exhaustion Epigenetic Programs during a Clinical Response

Caitlin C. Zebley, Charmaine Brown, Tian Mi, Yiping Fan, Shanta Alli, Shannon Boi, Giovanni Galletti, Enrico Lugli, Deanna Langfitt, Jean-Yves Metais, Timothy Lockey, Michael M Meagher, Brandon M Triplett, Aimee C Talleur, Stephen Gottschalk, Benjamin Youngblood
Journal: Blood
Blood (2021) 138 (Supplement 1): 2782.
https://doi.org/10.1182/blood-2021-151177
Published: 2021
Journal Articles

The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective

Nancy S. Bolous, Yichen Chen, Huiqi Wang, Andrew M. Davidoff, Meenakshi Devidas, Timothy W. Jacobs, Michael M. Meagher, Amit C. Nathwani, Ellis J. Neufeld, Bryan A. Piras, Carlos Rodriguez-Galindo, Ulrike M. Reiss, Nickhill Bhakta
Journal: Blood
Blood (2021) 138 (18): 1677–1690.
https://doi.org/10.1182/blood.2021010864
Published: 2021
Includes: Supplemental data
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

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Suk See De Ravin, MD, PhD, Sandra Anaya O'Brien, Nana Kwatemaa, RN, MSN, CCRP, Narda Theobald, BS, Siyuan Liu, Janet Lee, BS, Lela Kardava, Taylor Liu, Frederick Goldman, MDMS, Susan Moir, Jack Bleesing, MD PhD, Benedicte Neven, MD PhD, Jennifer Puck, MD, Morton J Cowan, MD, Ewelina Mamcarz, MD, Stephen Gottschalk, MD, Michael M Meagher, PhD, Luigi Notarangelo, MD, Elizabeth Kang, MD, Xiaolin Wu, PhD, Harry L. Malech, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 608.
https://doi.org/10.1182/blood-2019-127439
Published: 2019
Journal Articles

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Amit C Nathwani, MD PhD, Ulrike Reiss, MD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Anne Riddell, Jun Pie, Johnny N Mahlangu, MD, Michael Recht, MD PhD, Yu-Min Shen, MD, Kathleen G. Halka, MD, Michael M Meagher, PhD, Arthur W. Nienhuis, MD, Andrew M. Davidoff, MD, Sarah Mangles, Chris L Morton, Zhou Junfang, Vlad Calin Radulescu, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 491.
https://doi.org/10.1182/blood-2018-99-118334
Published: 2018
Journal Articles

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Amit C Nathwani, MD PhD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Doyoung Lee, PhD, Cecilia Rosales, PhD, Mark Phillips, Jun Pie, Zhou Junfang, Michael M Meagher, PhD, Ulrike Reiss, MD, Andrew M. Davidoff, MD, Chris L Morton, Anne Riddell
Journal: Blood
Blood (2018) 132 (Supplement 1): 489.
https://doi.org/10.1182/blood-2018-99-118256
Published: 2018
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