DÉLIVRANCE, UTILISATION ET APPLICATIONS THÉRAPEUTIQUES DES SYSTÈMES CRISPR-CAS ET COMPOSITIONS PERMETTANT DE CIBLER DES TROUBLES ET MALADIES AU MOYEN DE CONSTITUANTS DE DÉLIVRANCE SOUS FORME DE PARTICULES
The Broad Institute, Inc.; Inc.;Massachusetts Institute of Technology;The Broad Institute
发明人:
申请号：
EP14827606.6
公开号：
EP3079726B1
申请日:
2014.12.12
申请国别(地区):
EP
年份:
2018
代理人:
摘要：
The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery particle formulations and/or systems comprising one or more components of a CRISPR-Cas system, which are means for targeting sites for delivery. The delivery particle formulations of the invention are preferably nanoparticle delivery formulations and/or systems. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.
