Genome editing rescue of spinal muscular atrophy in cells and in mice
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下载链接：
https://www.ncbi.nlm.nih.gov/sra/SRP393039
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资源简介：
We describe one-time genome editing approaches targeting SMN2 that rescue native SMN protein levels and disease phenotypes in cell and mouse models of SMA.
创建时间：
2023-03-30
