Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9-Reference-Cited by-同舟云学术

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Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9

Published:2015-08 Issue:2 Volume:17 Page:213-220
ISSN:1934-5909
Container-title:Cell Stem Cell
language:en
Short-container-title:Cell Stem Cell

Author:

Park Chul-Yong,Kim Duk Hyoung,Son Jeong Sang,Sung Jin Jea,Lee Jaehun,Bae Sangsu,Kim Jong-Hoon,Kim Dong-Wook,Kim Jin-Soo

Publisher

Elsevier BV

Subject

Cell Biology,Genetics,Molecular Medicine

Reference33 articles.

1. Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases;Bae;Bioinformatics,2014

2. Int22h-related inversions causing hemophilia A: a novel insight into their origin and a new more discriminant PCR test for their detection;Bagnall;J. Thromb. Haemost.,2006

3. Inversion of the IDS gene resulting from recombination with IDS-related sequences is a common cause of the Hunter syndrome;Bondeson;Hum. Mol. Genet.,1995

4. Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors;Chao;Mol. Ther.,2001

5. Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease;Cho;Nat. Biotechnol.,2013

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