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application, manipulation and optimization of systems, methods and compositions for targeting and modeling post-mitotic cell diseases and disorders

机译:靶向和建模有丝分裂后细胞疾病和障碍的系统,方法和组合物的应用,操作和优化

摘要

Patent Summary for “Application, Handling, and Optimization of Systems, Methods, and Compositions for Targeting and Modeling Dog Diseases and Disorders” post-mitotic squids � the invention provides the application, manipulation and optimization of systems, methods, and compositions for manipulation of sequences and / or target sequence activities. application systems and tissues or organs are provided that include post-mitotic cells that are intended as application sites. Vectors and vector systems are also provided, some of which encode one or more components of a crispr complex, as well as methods for the design and use of these vectors. Also provided are methods of directing the formation of the crispr complex in eukaryotic cells to ensure increased specificity for target recognition and avoid toxicity and for editing or modifying a target site at a genomic site. Of interest to change or improve the condition of a disease or condition.
机译:有丝分裂鱿鱼后的“靶向和模拟狗疾病和障碍的系统,方法和成分的应用,处理和优化”的专利摘要。本发明提供了用于控制,控制和优化犬类疾病和病症的系统,方法和成分的应用,处理和优化。序列和/或目标序列活动。提供了包括打算用作应用部位的有丝分裂后细胞的应用系统和组织或器官。还提供了载体和载体系统,其中一些编码了保鲜盒复合物的一个或多个成分,以及设计和使用这些载体的方法。还提供了指导在真核细胞中形成保鲜盒复合物的方法,以确保增加对靶标识别的特异性并避免毒性以及在基因组位点编辑或修饰靶标位点。改变或改善疾病或状况的状态是令人感兴趣的。

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