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Nola Neri
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Journal Articles

Efficient prime editing of human hematopoietic stem cells corrects the recurrent SBDS C.258+2T>C mutation and recovers engraftment potential in shwachman-diamond syndrome

Free
Gue-Ho Hwang, Michael Peters, Felicia Andresen, Jing Zeng, Haarika Kathi, Sébastien Levesque, Chad Harris, Christy Kolsteeg, Nola Neri, Archana Verma, Eva Wauters, Chi Yuan Zhang, Myriam Armant, Christian Brendel, Scot Wolfe, John Manis, Akiko Shimamura, Daniel Bauer
Journal: Blood
Blood (2025) 146 (Supplement 1): 1063.
https://doi.org/10.1182/blood-2025-1063
Published: 2025
Journal Articles

Efficient, potent, and specific near-universal base and prime editing for ELANE-mutant severe congenital neutropenia

Free
Jing Zeng, Nola Neri, Josias Soares de Brito, Archana Verma, Sébastien Levesque, Gue-Ho Hwang, Viktoria Jung, Gabriele Casirati, Felicia Andresen, David Justus, Denisa Wagner, John Manis, Danilo Pellin, Pietro Genovese, Akiko Shimamura, Myriam Armant, Scot Wolfe, Peter Newburger, Daniel Bauer
Journal: Blood
Blood (2025) 146 (Supplement 1): 624.
https://doi.org/10.1182/blood-2025-624
Published: 2025
Journal Articles

Epitope Editing Combined with Extended Schedule Anti-KIT Antibody Treatment Allows Immune-Based In Vivo Selection of Multiplex Genome-Engineered Cells

Free
Gabriele Casirati, Adele Mucci, Marta Freschi, Andrea Cosentino, Mohammed S Mahmoud, Jing Zeng, Nola Neri, Christian Brendel, Daniel Bauer, Pietro Genovese
Journal: Blood
Blood (2024) 144 (Supplement 1): 4767.
https://doi.org/10.1182/blood-2024-202480
Published: 2024
Journal Articles

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

Free
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