Base editing as a genetic treatment for spinal muscular atrophy
收藏NIAID Data Ecosystem2026-05-01 收录
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资源简介：
This project aimed to develop a base editing approach to precisely edit SMN2. A range of different adenosine base editors (ABEs) and Cas9 enzymes were tested in HEK293T cells and a selected base editor was further tested in fibroblasts derived from patients with spinal muscular atrophy (SMA) and a SMA mouse model.
创建时间：
2023-09-09
