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ENGINEERED CRISPR/CAS9 SYSTEMS FOR SIMULTANEOUS LONG-TERM REGULATION OF MULTIPLE TARGETS

机译:用于同时长期调节多个目标的工程式CRISPR / CAS9系统

摘要

The invention provides CRISPR-based compositions and methods comprising non-repetitive sgRNA promoter and handle sequences for simultaneous, stable expression of multiple sgRNAs. In one embodiment, the invention relates to a nucleic acid molecule comprising an extra long sgRNA array (ELSA) for expression of at least two sgRNA sequences comprising: nucleotide sequences encoding two or more sgRNA sequence, wherein each sgRNA encoding nucleotide sequence is under the control of a sgRNA promoter and operably linked to a sgRNA handle sequence; wherein the ELSA comprises a maximum shared repeat length of 20 nucleotides or less. In one embodiment, the ELSA comprises a maximum shared repeat length 12 nucleotides or less.
机译:本发明提供了基于CRISPR的组合物和方法,包括非重复的SGRNA启动子和用于同时稳定的多个SGRNA表达的手柄序列。在一个实施方案中,本发明涉及包含超长SGRNA阵列(ELSA)的核酸分子,用于表达至少两个SGRNA序列,所述SGRNA序列包括编码两种或更多种SGRNA序列的核苷酸序列,其中编码核苷酸序列的每个SGRNA在控制下SGRNA启动子和与SGRNA手柄序列可操作地连接;其中,ELSA包含20个核苷酸或更小的最大共同重复长度。在一个实施方案中,ELSA包括最大共同的重复长度12个核苷酸或更少。

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