Targeting RNA opens therapeutic avenues for Timothy syndrome
成果类型：
Editorial Material
署名作者：
Velasco, Silvia
署名单位：
Murdoch Children's Research Institute
刊物名称：
Nature
ISSN/ISSBN：
0028-3733
DOI：
10.1038/d41586-024-00911-1
发表日期：
2024-04-25
页码：
730-732
关键词：
摘要：
A therapeutic strategy that alters gene expression in a rare and severe neurodevelopmental condition has been tested in stem-cell-based models of the disease, and has been shown to correct genetic and cellular defects.
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