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John P Manis, MD
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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

Therapeutic Base Editing of Human Hematopoietic Stem Cells

Jing Zeng, MD, Yuxuan Wu, PhD, Chunyan Ren, PhD, Jasmine Bonanno, Anne H. Shen, Devlin Shea, Jason M Gehrke, PhD, John P Manis, MD, J. Keith Gehrke, Daniel E. Bauer, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 612.
https://doi.org/10.1182/blood-2019-129173
Published: 2019
Journal Articles

Highly Efficient Therapeutic Gene Editing of BCL11A enhancer in Human Hematopoietic Stem Cells from ß-Hemoglobinopathy Patients for Fetal Hemoglobin Induction

Jing Zeng, MD, Daniel E. Bauer, MD PhD, Yuxuan Wu, PhD, Benjamin P Roscoe, Pengpeng Liu, Qiuming Yao, PhD, Cicera Lazzarrotto, M. Kendell Clement, Mitchel A. Cole, BS, Kevin Luk, Cristina Baricordi, Erica B. Esrick, MD, John P Manis, MD, David Dorfma, David A. Williams, MD, Alessandra Biffi, MD PhD, Carlo Brugnara, MD, Luca Biasco, Christian Brendel, PhD, Luca Pinello, PhD, Shengdar Tsai, PhD, Scot A. Wolfe
Journal: Blood
Blood (2018) 132 (Supplement 1): 3482.
https://doi.org/10.1182/blood-2018-99-119365
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