DELIVERY, CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND USE IN THERAPY

摘要

FIELD: biotechnology.;SUBSTANCE: methods are proposed for modifying a eukaryotic organism by manipulating a target sequence in a genome locus of interest by using the CRISPR-Cas system, as well as a method of treating or inhibiting a condition caused by a defect in a target sequence, and a composition containing components of said system.;EFFECT: present invention enables the change of genomic loci of the eukaryotic cell by using Cas9 nuclease fused with one or more nuclear localization sequences (NLS).;21 cl, 73 dwg, 35 tbl, 37 ex