Article

Amelioration of hemophilia B through CRISPR/Cas9 induced homology-independent targeted integration

2021-03-18

Abstract excerpt

<title>Abstract</title> <p>Site-specific integration of exogenous gene through genome editing is a promising strategy for gene therapy. However, homology-directed repair (HDR) only occurring in proliferating cells is inefficient especially in vivo. To investigate the efficacy of Cas9-induced homology-independent targeted integration (HITI) strategy for gene therapy, a rat hemophilia B model was generated and empl...

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Literature Corpus work: 51834f12-0cd3-5bf6-8e42-b4166f35b66c
DOI: 10.21203/rs.3.rs-302699/v1

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