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Journal Articles

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

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

Blood (2025) 146 (Supplement 1): 624.

https://doi.org/10.1182/blood-2025-624

Published: 2025

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

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

Blood (2025) 146 (Supplement 1): 1063.

https://doi.org/10.1182/blood-2025-1063

Published: 2025