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CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice

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NIAID Data Ecosystem2026-03-12 收录
下载链接:
https://www.ncbi.nlm.nih.gov/sra/SRP299165
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资源简介:
This study provides compelling evidence to further investigate editing of the homologous region of the human UBE3A-ATS, since this may provide a lasting therapeutic effect for AS patients.
创建时间:
2020-12-24
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