High-efficiency genetic correction of primary hyperoxaluria type 1 causing AGXT variant, c.508G>A (Gly170Arg) with an adenine base editor in patient-derived fibroblasts

被引:0
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作者
Keskinen, T. [1 ]
Jalil, S. [1 ]
Hyvonen, M. E. [1 ,2 ]
Wartiovaara, K. [1 ,2 ]
机构
[1] Univ Helsinki, Helsinki, Finland
[2] Helsinki Univ Hosp, Helsinki, Finland
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中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
P586
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页码:A250 / A250
页数:1
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    MOLECULAR THERAPY, 2019, 27 (04) : 242 - 242