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Marioara Felicia Ciuculescu
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Journal Articles

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

Erica B. Esrick, Amy Federico, Daniela Abriss, Myriam Armant, Kari Boardman, Christian Brendel, Marioara-Felicia Ciuculescu, Heather Daley, Colleen Dansereau, Augustine Fernandes, Matthew M. Heeney, David G. Justus, Pei-Chi Kao, Annette S. Kim, Donald B. Kohn, Leslie E. Lehmann, Donghui Liu, Wendy B. London, John P Manis, Theodore B. Moore, Emily Morris, Danilo Pellin, Ellen Proeung, Shanna Richard, Gavin D. Roach, Kit L. Shaw, Dayna Terrazas, Shanna L White, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10665–10667.
https://doi.org/10.1182/blood-2022-169089
Published: 2022
Journal Articles

Therapeutic Gene Editing of HSCs Ex Vivo without in Vitro Culture Avoids Genotoxicity, Simplifies Procedures, and Preserves Efficiency and Stemness

Jing Zeng, My Anh Nguyen, Marioara-Felicia Ciuculescu, Esther Mintzer, Pengpeng Liu, Linda Yingqi Lin, Tolulope O Rosanwo, Archana Verma, Nola Neri, Stacy A. Maitland, Alden Richter, David G. Justus, Kendell Clement, Christian Brendel, Luca Pinello, John P Manis, Myriam Armant, Scot A. Wolfe, Daniel E. Bauer
Journal: Blood
Blood (2022) 140 (Supplement 1): 10671–10672.
https://doi.org/10.1182/blood-2022-169900
Published: 2022
Journal Articles

Human Genetic Diversity Alters Therapeutic Gene Editing Off-Target Outcomes

Linda Yingqi Lin, Samuele Cancellieri, Jing Zeng, Francesco Masillo, My Anh Nguyen, Nicola Bombieri, Stacy A. Maitland, Marioara-Felicia Ciuculescu, Varun Katta, Shengdar Q. Tsai, Myriam Armant, Scot A. Wolfe, Rosalba Giugno, Luca Pinello, Daniel E. Bauer
Journal: Blood
Blood (2021) 138 (Supplement 1): 3993.
https://doi.org/10.1182/blood-2021-152429
Published: 2021
Journal Articles

Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models

Jing Zeng, Selami Demirci, My Anh Nguyen, Linda Yingqi Lin, Stacy A. Maitland, Esther Mintzer, Yuxuan Wu, Danilo Pellin, Amornrat Tangprasittipap, Chokdee Vong, Shaina N. Porter, Kevin Luk, Pengpeng Liu, Varun Katta, Marioara-Felicia Ciuculescu, Daniela Abriss, Jonathan Hsu, Naoya Uchida, Khaled Essawi, Robert Donahue, Karl Petri, Vikram Pattanayak, Luca Pinello, Christian Brendel, David A Williams, John P Manis, Shengdar Q. Tsai, Shondra M. Pruett-Miller, J. Keith Joung, Duantida Songdej, Suradej Hongeng, Myriam Armant, Scot A. Wolfe, John F. Tisdale, Daniel E. Bauer
Journal: Blood
Blood (2021) 138 (Supplement 1): 1852.
https://doi.org/10.1182/blood-2021-152634
Published: 2021
Journal Articles

Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Distributions of HbF in Single-RBCs and Reticulocytes

Nicolas Hebert, Erica B. Esrick, Myriam Armant, Christian Brendel, Marioara Felicia Ciuculescu, Etienne Audureau, John M. Higgins, David A. Williams, Pablo Bartolucci
Journal: Blood
Blood (2021) 138 (Supplement 1): 967.
https://doi.org/10.1182/blood-2021-150186
Published: 2021
Journal Articles

Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal Hemoglobin Using Post-Transcriptional Gene Silencing

Erica B. Esrick, MD, Maureen Achebe, MD MPH, Myriam Armant, PhD, Pablo Bartolucci, MD PhD, Marioara Felicia Ciuculescu, MD, Heather Daley, Colleen Dansereau, MSN, RN, CPN, Giuseppe Di Caprio, PhD, Bronner Goncalves, MD PhD, Nicolas Hebert, Matthew M Heeney, MD, John M. Higgins, MD PhD, Leslie E. Lehmann, MD, John P Manis, MD, Olivier Negre, PhD, Sarah Nikiforow, MD PhD, Ethan Schonbrun, PhD, David K. Wood, PhD, David A. Williams, MD
Journal: Blood
Blood (2019) 134 (Supplement_2): LBA-5.
https://doi.org/10.1182/blood-2019-132745
Published: 2019
Journal Articles

Deletion of Murine Rhoh induces More Aggressive Diffuse Large B Cell Lymphoma (DLBCL) Via Interaction with Kaiso and Regulation of BCL-6 Expression

Hiroto Horiguchi, MDPhD, Marioara Felicia Ciuculescu, MD, Anja Troeger, MD, Haiming Xu, PhD, Christian Brendel, PhD, David A. Williams, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 1574.
https://doi.org/10.1182/blood-2018-99-115238
Published: 2018
Journal Articles

Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients

Erica B. Esrick, MD, Christian Brendel, PhD, John P Manis, MD, Myriam A Armant, PhD, Helene Negre, PhD, Colleen Dansereau, MSN, RN, CPN, Marioara Felicia Ciuculescu, MD, Stephanie Patriarca, Brenda Mackinnon, BSN, RN, Heather Daley, BS, Shanna Richard, Daniela Abriss, Renee Maxwell, Stephen Braunewell, BS, Lauryn Christiansen, BS, Sarah Nikiforow, MD PhD, Jerome Ritz, MD PhD, Maureen Achebe, MD, Olivier Negre, PhD, Leslie E. Lehmann, MD, Matthew M Heeney, MD, Alessandra Biffi, MD PhD, David A. Williams, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 1023.
https://doi.org/10.1182/blood-2018-99-116733
Published: 2018
Journal Articles

Deletion of Rhoh Accelerates Development of Diffuse Large B Cell Lymphoma in Bcl-6 Transgenic Mouse Model

Hiroto Horiguchi, MDPhD, Marioara Felicia Ciuculescu, MD, Christian Brendel, PhD, David A. Williams, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 1470.
https://doi.org/10.1182/blood.V130.Suppl_1.1470.1470
Published: 2017