首页> 外国专利> METHODS FOR EDITING GENES AND COMPOSITIONS TO REDUCE THE RISK OF ACTIVATION OF THE JC VIRUS AND PML (PROGRESSING MULTIFOCAL LEU-ENCEPHALOPATHY) DURING IMMUNOSUPRESSIVE THERAPY

METHODS FOR EDITING GENES AND COMPOSITIONS TO REDUCE THE RISK OF ACTIVATION OF THE JC VIRUS AND PML (PROGRESSING MULTIFOCAL LEU-ENCEPHALOPATHY) DURING IMMUNOSUPRESSIVE THERAPY

机译:编辑基因和成分的方法以降低免疫抑制治疗期间JC病毒和PML(进行性多灶性白质脑病)的激活风险

摘要

A method of eliminating the risk of JCV activation in a subject undergoing immunosuppressive therapy, by administering an effective amount of a gene editing composition directed toward at least one target sequence in the JCV genome, cleaving the target sequence in the JCV genome, disrupting the JCV genome, eliminating the JCV infection, eliminating the risk of JCV activation, and treating the subject with an immunosuppressive therapy. A pharmaceutical composition including at least one isolated nucleic acid sequence encoding a CRISPR-associated endonuclease and at least one gRNA having a spacer sequence complementary to a target sequence in a JCV DNA, the isolated nucleic acid sequences being included in at least one expression vector. Pharmaceutical compositions including at least one isolated nucleic acid sequence encoding at least one TALEN, at least one ZFN, and gene editing composition of C2c1, C2c3, TevCas9, Archaea Cas9, CasY.1-CasY.6, CasX, or argonaute protein, which target at least one nucleotide sequence of the JCV genome.
机译:一种通过给予有效量的针对JCV基因组中至少一个靶序列的基因编辑组合物,切割JCV基因组中的靶序列,破坏JCV来消除进行免疫抑制疗法的受试者中JCV激活的风险的方法基因组,消除JCV感染,消除JCV活化的风险,并用免疫抑制疗法治疗对象。药物组合物,其包含至少一种编码CRISPR相关核酸内切酶的分离的核酸序列和至少一种具有与JCV DNA中的靶序列互补的间隔区序列的gRNA,所述分离的核酸序列包含在至少一种表达载体中。药物组合物,其包含至少一种分离的核酸序列,其编码至少一种TALEN,至少一种ZFN以及C2c1,C2c3,TevCas9,古细菌Cas9,CasY.1-CasY.6,CasX或精氨酸蛋白的基因编辑组合物,靶向JCV基因组的至少一个核苷酸序列。

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