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Giuseppe Ciaramella
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Journal Articles

Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises

Ashish O. Gupta, Akshay Sharma, Haydar Frangoul, Jignesh Dalal, Julie Kanter, Asif Alavi, John DiPersio, Mary Eapen, Jennifer J. Jaroscak, Ernesto Ayala, Edward D. Ziga, Stacey Rifkin-Zenenberg, Alex C. Minella, Guo Chen, Yinzhong Chen, Priya S. Chockalingam, Ling Lin, Marcelyne Joseney-Antoine, Leanne Ianniello, Beth Gardner, Adam J. Hartigan, Giuseppe Ciaramella, Sunita Goyal, Amy Simon, Alexis Thompson, Matthew M. Heeney
Journal: Blood
Blood (2024) 144 (Supplement 1): 513.
https://doi.org/10.1182/blood-2024-204888
Published: 2024
Journal Articles

Evaluation of the Impact of a Naturally Occurring Beta Hemoglobin Variant, Hb G-Makassar, on Mature Red Blood Cell Function and Pathology in a Sickle Cell Disease Mouse Model

Zachary Kostamo, Jawa Darazim, Britney Hernandez, Alex Pendergast, Jordan Zgodny, Erica N Evans, Yankai Zhang, Ashwin P Patel, Celeste K. Kanne, Elizabeth Budak, Rebecca Jenkins, Daniel Haupt, Valerie Winton, Bo Yan, Adam Hartigan, Haihua Chu, Giuseppe Ciaramella, Vivien A. Sheehan
Journal: Blood
Blood (2023) 142 (Supplement 1): 2251.
https://doi.org/10.1182/blood-2023-186237
Published: 2023
Journal Articles

Non-Genotoxic Conditioning for Hematopoietic Stem Cell Transplant through Engineered Stem Cell Antibody Paired Evasion (ESCAPE)

Nandini Mondal, Alexander Harmon, Elizabeth Budak, Kathy Zhang, Jeffrey Wong, Archita Venugopal Menon, Adam Wolin, Moriah White, Faith Musenge, Adam Camblin, Brent Coisman, Corrina Lucini, Tao Bai, Kangjian Qiao, Wayne Austin, Michelle DeLelys, Hugh Kromer, Lisa Hardy, Megan Law, Raffi Manoukian, Rachel Goldsmith, Kiera Fallon, Mudra Patel, Antoinette Cozier, Colin Lazzara, Jeremy Decker, Rebecca Jenkins, Tanggis Bohnuud, Patricia Feliciano, Seung-Joo Lee, Paul Kopesky, Sarah Smith, Charlotte McDonagh, Nicole Gaudelli, S. Haihua Chu, Adam Hartigan, Gopi Shankar, Giuseppe Ciaramella
Journal: Blood
Blood (2023) 142 (Supplement 1): 7128.
https://doi.org/10.1182/blood-2023-182077
Published: 2023
Journal Articles

Engineered Stem Cell Antibody Paired Evasion-2 (ESCAPE-2): Paired HSC Epitope Engineering and Direct Editing of Sickle Allele for Antibody-Mediated Autologous Hematopoietic Stem Cell Therapy Conditioning for the Treatment of Sickle Cell Disease

S. Haihua Chu, Elizabeth Budak, Nandini Mondal, Kathy Zhang, Alexander Harmon, Jeffrey Wong, Dieter Lam, Corrina Lucini, Brent Coisman, Kangjian Qiao, Tao Bai, Jeremy Decker, Bob Gantzer, Kindo Lee, Maya Sen, Tanggis Bohnuud, Luis Barrera, Brian Cafferty, Seung-Joo Lee, Paul Kopesky, Nicole M. Gaudelli, Charlotte F McDonagh, Giuseppe Ciaramella, Adam J. Hartigan
Journal: Blood
Blood (2022) 140 (Supplement 1): 10213–10214.
https://doi.org/10.1182/blood-2022-169257
Published: 2022
Journal Articles

Engineered Stem Cell Antibody Paired Evasion 1 (ESCAPE-1): Paired HSC Epitope Engineering and Upregulation of Fetal Hemoglobin for Antibody-Mediated Autologous Hematopoietic Stem Cell Therapy Conditioning for the Treatment of Hemoglobinopathies

Nandini Mondal, Alexander Harmon, Elizabeth Budak, Kathy Zhang, Jeffrey Wong, Conrad Rinaldi, Jenny Olins, Kara Hoar, Archita Venugopal Menon, Moriah White, Faith Musenge, Adam Camblin, Adam Wolin, Brent Coisman, Tao Bai, Kangjian Qiao, Mudra Patel, Jeremy Decker, Bob Gantzer, Thomas Leete, Yi Yu, Tanggis Bohnuud, Seung-Joo Lee, Sarah Smith, Charlotte F McDonagh, Nicole M. Gaudelli, S. Haihua Chu, Adam J. Hartigan, Giuseppe Ciaramella
Journal: Blood
Blood (2022) 140 (Supplement 1): 4487–4488.
https://doi.org/10.1182/blood-2022-169120
Published: 2022
Journal Articles

Cytosine base editing enables quadruple-edited allogeneic CART cells for T-ALL

Caroline Diorio, Ryan Murray, Mark Naniong, Luis Barrera, Adam Camblin, John Chukinas, Lindsey Coholan, Aaron Edwards, Tori Fuller, Claudia Gonzales, Stephan A. Grupp, Alden Ladd, Melissa Le, Angelica Messana, Faith Musenge, Haley Newman, Yeh-Chuin Poh, Henry Poulin, Theresa Ryan, Rawan Shraim, Sarah K. Tasian, Tiffaney Vincent, Lauren Young, Yingying Zhang, Giuseppe Ciaramella, Jason Gehrke, David T. Teachey
Journal: Blood
Blood (2022) 140 (6): 619–629.
https://doi.org/10.1182/blood.2022015825
Published: 2022
Includes: Supplemental data
Journal Articles

Conversion of HbS to Hb G-Makassar By Adenine Base Editing Is Compatible with Normal Hemoglobin Function

S. Haihua Chu, Manuel Ortega, Paty Feliciano, Valerie Winton, Chavonna Xu, Daniel Haupt, Teresa McDonald, Salette Martinez, Alexander Liquori, Jeffrey Marshall, Daisy Lam, Jenny Olins, Conrad Rinaldi, Kyle Rehberger, Colin Lazarra, Jeremy Decker, Bob Gantzer, Tanggis Bohnuud, David Born, Luis Barrera, Bo Yan, Ian Slaymaker, Michael S. Packer, Sarah Smith, Carlo Zambonelli, Matt Lee, Nicole M. Gaudelli, Adam J. Hartigan, Giuseppe Ciaramella
Journal: Blood
Blood (2021) 138 (Supplement 1): 951.
https://doi.org/10.1182/blood-2021-150922
Published: 2021
Journal Articles

Screening of Chemically Distinct Lipid Nanoparticles In Vivo Using DNA Barcoding Technology Towards Effectively Delivering Messenger RNA to Hematopoietic Stem and Progenitor Cells

Cory Sago, Elizabeth Campbell, Brianna Lutz, Neeraj Patwardhan, Gregory Hamilton, Courtney Wong, Daniel Lee, Shayon Keating, Kathryn Murray, Manmohan Singh, Giuseppe Ciaramella
Journal: Blood
Blood (2021) 138 (Supplement 1): 2931.
https://doi.org/10.1182/blood-2021-153652
Published: 2021
Journal Articles

Adenine Base Editing of the Sickle Allele in CD34 + Hematopoietic Stem and Progenitor Cells Eliminates Hemoglobin S

S. Haihua Chu, PhD, Daisy Lam, Michael S. Packer, PhD, Jennifer Olins, Alexander Liquori, Jeffrey Marshall, PhD, Calvin Lee, Bo Yan, PhD, Jeremy Decker, Bob Gantzer, M.S., Scott J. Haskett, M.S., Tanggis Bohnuud, PhD, David Born, PhD, Luis Barrera, PhD, Ian Slaymaker, PhD, Nicole M. Gaudelli, PhD, Adam J. Hartigan, PhD, Giuseppe Ciaramella, PhD, B.Sc.
Journal: Blood
Blood (2020) 136 (Supplement 1): 47.
https://doi.org/10.1182/blood-2020-141805
Published: 2020
Journal Articles

Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and Beta Thalassemia

Ling Lin, PhD, Adrian P. Rybak, PhD, Conrad Rinaldi, M.S., Jonathan Yen, PhD, Yanfang Fu, PhD, Elsie Akrawi, Sarah Smith, Scott J. Haskett, M.S., Minerva E. Sanchez, Yeh-Chuin Poh, PhD, Michael S. Packer, PhD, Nicole M. Gaudelli, PhD, Manmohan Singh, PhD, Giuseppe Ciaramella, PhD, B.Sc., Dana N. Levasseur, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3352.
https://doi.org/10.1182/blood-2019-126710
Published: 2019
Journal Articles

Highly Efficient Multiplexed Base Editing with Minimized Off-Targets for the Development of Universal CAR-T Cells to Treat Pediatric T-ALL

Jason M Gehrke, PhD, Aaron Edwards, MS, Ryan C. Murray, MS, Amy Shaw, MS, Yeh-Chuin Poh, PhD, Sarah Smith, Yi Yu, PhD, Nicole M. Gaudelli, PhD, Waseem Qasim, MBBS, PhD, Giuseppe Ciaramella, PhD, B.Sc.
Journal: Blood
Blood (2019) 134 (Supplement_1): 5127.
https://doi.org/10.1182/blood-2019-130562
Published: 2019