CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs

Volume: 111, Issue: 2, Pages: 225 - 233
Published: Oct 29, 2019
Abstract
The clustered regulatory interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system is an efficient genome-editing tool that holds potential for gene therapy. Here, we report an application of this system for gene repair in hemophilia B (HB) using induced pluripotent stem cells (iPSCs). We prepared targeting plasmids with homology arms containing corrected sequences to repair an in-frame deletion in exon 2 of the factor IX...
Paper Details
Title
CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs
Published Date
Oct 29, 2019
Volume
111
Issue
2
Pages
225 - 233
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