首页> 外国专利> USE AND PRODUCTION OF CHD8+/- TRANSGENIC ANIMALS WITH BEHAVIORAL PHENOTYPES CHARACTERISTIC OF AUTISM SPECTRUM DISORDER

USE AND PRODUCTION OF CHD8+/- TRANSGENIC ANIMALS WITH BEHAVIORAL PHENOTYPES CHARACTERISTIC OF AUTISM SPECTRUM DISORDER

机译:具有自发性光谱障碍行为表型特征的CHD8 +/-转基因动物的使用和生产

摘要

The invention involves inducing a plurality e.g., 3-50 or more mutations (e.g., any whole number between 3 and 50 or more of mutations, with it noted that in some embodiments there can be up to 16 different RNA(s), e.g., sgRNAs each having its own a promoter, in a vector, such an AAV vector or a lentiviral vector and that when each sgRNA does not have its own promoter, there can be twice to thrice that amount of different RNA(s), e.g., sgRNAs, e.g., 32 or even 48 different guides delivered by one vector) in transgenic Cas9 eukaryotes to model a neuronal disease or disorder. The invention comprehends testing putative treatments with such models, e.g., testing putative chemical compounds that may be pharmaceutically relevant for treatment or gene therapy that may be relevant for treatment, or combinations thereof. The invention allows for the study of genetic diseases and putative treatments to better understand and alleviate a genetic disease or a condition, e.g., autism, autism-spectrum disease or disorder, obsessive compulsive disorder, or psychiatric disorders.
机译:本发明涉及诱导多个例如3-50或更多个突变(例如3至50或更多个突变之间的任何整数,要注意的是,在一些实施方案中,可以存在多达16个不同的RNA,例如, sgRNA在载体(例如AAV载体或慢病毒载体)中各自具有自己的启动子,并且当每个sgRNA不具有其自身的启动子时,可以有两倍于此数量的不同RNA,例如sgRNA (例如,由一种载体递送的32种或什至48种不同的向导)在转基因Cas9真核生物中来模拟神经元疾病或病症。本发明包括用这样的模型测试推定的治疗,例如,测试与治疗可能在药物上相关的推定的化合物或与治疗有关的基因疗法,或其组合。本发明允许对遗传疾病和推定疗法进行研究,以更好地理解和减轻遗传疾病或病症,例如自闭症,自闭症谱系疾病或病症,强迫症或精神病。

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