Editing aberrant splice sites efficiently restores Beta-globin expression in Beta-thalassemia
收藏NIAID Data Ecosystem2026-03-10 收录
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资源简介：
Aberrant HBB splice sites were disrupted to restore gene function in CD34+ hematopoietic stem cells from Beta-thalassemia patient donors. The IVS1-110G>A mutation was targeted with Cas9 RNP and the IVS2-654C>T mutation with Cas12a/Cpf1 RNP.
创建时间：
2019-01-29
