DELIVERY, USE AND THERAPEUTIC APPLICATIONS OF THE

专利名称：DELIVERY, USE AND THERAPEUTIC

APPLICATIONS OF THE CRISPR-CASSYSTEMS AND COMPOSITIONS FORTARGETING DISORDERS AND DISEASESUSING PARTICLE DELIVERY COMPONENTS

发明人：PLATT, Randall Jeffrey,LANGER,

Robert,ZHANG, Feng,ANDERSON,Daniel,DAHLMAN, James

申请号：EP18199415.3申请日：20141212公开号：EP3470089A1公开日：20190417

专利附图：

摘要：The invention provides for delivery, engineering and optimization of systems,methods, and compositions for manipulation of sequences and/or activities of targetsequences. Provided are delivery particle formulations and/or systems comprising one ormore components of a CRISPR-Cas system, which are means for targeting sites fordelivery. The delivery particle formulations of the invention are preferably nanoparticledelivery formulations and/or systems. Also provided are vectors and vector systemssome of which encode one or more components of a CRISPR complex, as well as methodsfor the design and use of such vectors. Also provided are methods of directing CRISPRcomplex formation in eukaryotic cells to ensure enhanced specificity for targetrecognition and avoidance of toxicity and to edit or modify a target site in a genomiclocus of interest to alter or improve the status of a disease or a condition.

申请人：The Broad Institute Inc.,Massachusetts Institute Of Technology

地址：415 Main Street Cambridge, MA 02142 US,77 Massachusetts Avenue Cambridge,MA 02139 US

国籍：US,US

代理机构：Williams, Gareth Owen

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