Targeted inhibition of ELANE expression using adenine base editing to treat severe congenital neutropenia.
利用腺嘌呤碱基编辑靶向抑制 ELANE 表达来治疗重度先天性中性粒细胞减少症。
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2025.101626
Findik Betül, Dannenmann Benjamin, Bernhard Franka, Kaufmann Masako Monika, Ammann Sandra, Kandabarau Sergey, Klimiankou Maksim, Mauch Fabian, Münzer Patrick, Borst Oliver, Klefenz Isabel, Steinemann Doris, Lengerke Claudia, Zeidler Cornelia, Cathomen Toni, Welte Karl, Nasri Masoud, Skokowa Julia
