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".
Am J Med Genet A.2020 Feb ; 182(2):275-276.PubMedで表示
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PMID: 31943709 [PubMed - in process]