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Article

<i>Ex vivo</i> therapeutic base and prime editing using chemically derived hepatic progenitors in a mouse model of tyrosinemia type 1

2020-09-15

Abstract excerpt

<h4>Summary</h4> DNA base editors and prime editing technology capable of therapeutic base conversion enable ex vivo gene editing therapy for various genetic diseases. For such therapy, it is critical that the target cells survive well both outside the body and after transplantation. In this regard, chemically derived stem/progenitor cells are attracting attention as the most useful cell sources for clinical tri...

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Literature Corpus work
c378b658-60c3-5c76-8c0e-0ab4a78d1e47
DOI
10.1101/2020.09.14.297275
Open publication

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