Delivery, use and therapeutic applications of CRISPR-CAS systems and compositions for targeting disorders and diseases using particle delivery components

摘要

The present invention provides delivery, engineering and optimization of systems, methods and compositions for manipulating the sequence and / or activity of a target sequence. Delivery particle formulations and / or systems comprising one or more components of the CRISPR-Cas system that are means for targeting the delivery site are provided. The delivery particle formulation of the present invention is preferably a nanoparticle delivery formulation and / or system. Also provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, and methods for designing and using such vectors. Also, CRISPR complex in eukaryotic cells to ensure increased specificity for target recognition and avoid toxicity, and to edit or modify the target site at the target genomic locus to change or improve disease state or condition A method of inducing body formation is also provided. [Selection] Figure 1