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DELIVERY, USE AND THERAPEUTIC APPLICATIONS OF THE CRISPR-CAS SYSTEMS AND COMPOSITIONS FOR TARGETING DISORDERS AND DISEASES USING PARTICLE DELIVERY COMPONENTS

机译:CRISPR-CAS系统和组合物的递送,使用和治疗学应用,使用颗粒递送成分靶向疾病和疾病

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.
机译:本发明提供了用于操纵靶序列的序列和/或活性的系统,方法和组合物的递送,工程设计和优化。提供包含CRISPR-Cas系统的一种或多种组分的递送颗粒制剂和/或系统,其是靶向递送部位的手段。本发明的递送颗粒制剂优选是纳米颗粒递送制剂和/或系统。还提供了载体和载体系统,其中一些编码CRISPR复合物的一种或多种组分,以及设计和使用这种载体的方法。还提供了指导在真核细胞中形成CRISPR复合物的方法,以确保增强的靶标识别特异性和避免毒性,并编辑或修饰目的基因组位点中的靶位点,以改变或改善疾病或病症的状态。

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