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CRISPR comes of age: A fairytale turned into bedside reality?

CRISPR comes of age: A fairytale turned into bedside reality?

Fei Li
Jim Hu
Genes & Diseases第2卷, 第4期pp.284-285纸质出版 2015-12-01在线发表 2015-09-16
134102

We devote this short piece to highlight one recent article published in Cell Stem Cell, reporting the correction of large chromosomal inversions of the factor VIII (F8) gene in cells from Hemophilia A patients using the CRISPR-Cas9 technology, one of the first attempts to edit large segments of chromosomes in patient cells using such methodology. The corrected cells were found free of off-target mutations and producing functional factor VIII in hemophilia mouse model. This work heralds another major advance in bringing CRISPR closer to the therapeutic reality.

Chromosomal inversionsCRISPR-Cas9Endothelial cellsHemophilia AiPSCs