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Gene Targeting

Highly Efficient Gene Editing of Cystic Fibrosis Patient-derived Airway Basal Cells Results in Functional CFTR Correction
May 7th, 2020
Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets
May 7th, 2020
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
May 7th, 2020
Enhancing gene editing specificity by attenuating DNA cleavage kinetics
December 6th, 2019