首页> 外文期刊>American journal of medical genetics, Part A >Mucopolysaccharidosis Type I Phenotypically Corrected with Edited Hematopoietic Stem Cells: Instead of altering the IDUA gene, a protein was inserted in a repurposable place in the genome known as a 'safe harbor locus'
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Mucopolysaccharidosis Type I Phenotypically Corrected with Edited Hematopoietic Stem Cells: Instead of altering the IDUA gene, a protein was inserted in a repurposable place in the genome known as a 'safe harbor locus'

机译:用编辑的造血干细胞表型I型I型典型:代替改变idua基因,将蛋白质插入所谓的“安全港口基因座”的基因组中的可重复的位置。

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