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Chi Yuan Zhang
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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

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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

Targeting a Putative Intronic Splicing Silencer Salvages Expression from the Recurrent SBDS C.258+2T>C Mutant Allele in Shwachman-Diamond Syndrome Patient Cells and Mouse Model

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Michael J. Peters, Anna Deck, April Hu, Yuko Fujiwara, Jinkuk Kim, Aubrie Soucy, Kevin Zhang, Julianna Kenny, Linda Y. Lin, Alyssa L. Kennedy, Caitlin Bowers, Chad E. Harris, Chi Yuan Zhang, Karthik Ponnienselvan, Pengpeng Liu, Kevin Luk, Davide Seruggia, R. Coleman Lindsley, John P. Manis, Christian Brendel, Scot A. Wolfe, Stuart H. Orkin, Timothy Yu, Akiko Shimamura, Daniel E. Bauer
Journal: Blood
Blood (2022) 140 (Supplement 1): 2944–2945.
https://doi.org/10.1182/blood-2022-169379
Published: 2022