학술논문
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
Document Type
Abstract
Author
Peters, Michael J. ; Deck, Anna ; Hu, April ; Fujiwara, Yuko ; Kim, Jinkuk ; Soucy, Aubrie ; Zhang, Kevin ; Kenny, Julianna ; Lin, Linda Y. ; Kennedy, Alyssa L.; Bowers, Caitlin ; Harris, Chad E. ; Zhang, Chi Yuan ; Ponnienselvan, Karthik ; Liu, Pengpeng ; Luk, Kevin ; Seruggia, Davide ; Lindsley, R. Coleman; Manis, John P. ; Brendel, Christian ; Wolfe, Scot A. ; Orkin, Stuart H.; Yu, Timothy ; Shimamura, Akiko; Bauer, Daniel E.
Source
In Blood 15 November 2022 140 Supplement 1:2944-2945
Subject
Language
ISSN
0006-4971