CRISPR Jump-Starts Gene Therapy

Abstract

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References

Cruz

et al. Organoid cystogenesis reveals a critical role of microenvironment in human polycystic kidney disease. Nat. Mater. , 2017; 16(11): 1112–1119. doi: 10.1038/nmat4994.

Sürün

et al. High-Efficiency Gene Correction in Hematopoietic Cells by Donor-Template-Free CRISPR/Cas9 Genome Editing. Mol. Ther. Nucleic Acids , 2018; 10: 1–8.

Osborn

et al. CRISPR/Cas9-Based Cellular Engineering for Targeted Gene Overexpression. Int. J. Mol. Sci. , 2018; 19: 946. doi: 10.3390/ijms19040946.

Akcakaya

et al. In vivo CRISPR-Cas gene editing with no detectable genome-wide off-target mutations. Nature , 2018; 561: 416–419.

Tsai

et al. CIRCLE-seq: A highly sensitive in vitro screen for genome-wide CRISPR-Cas9 nuclease off-targets. Nat. Methods , 2017; 14: 607–614.