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Paper Details

CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX-knockout mice.
Blood
50
2019
AAV vector genome, CRISPR, Cas9, FIX, FIX Padua mutation, clustered regularly interspaced short palindromic repeats, factor IX, genetic diseases, hemophilia, hepatocyte, human, human factor IX, mice, nonintegrating, promoterless partial human factor IX (FIX) complementary DNA
Author NameAffiliation
Mingyao LiUniversity of Pennsylvania
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Datasets

Release 1.2