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TARGETED TREATMENT OF AUTISM SPECTRUM DISORDER AND OTHER NEUROLOGICAL OR PSYCHIATRIC DISORDERS
TARGETED TREATMENT OF AUTISM SPECTRUM DISORDER AND OTHER NEUROLOGICAL OR PSYCHIATRIC DISORDERS
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机译:针对性治疗自闭症谱系疾病和其他神经或精神疾病
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摘要
Disclosed are methods of treating autism spectrum disorder (ASD) by administering a therapeutically effective amount of an isoprenoid antibiotic to subjects identified with a splicing defect in an ASD associated gene. The method of treating a subject with a neurological disease is carried out by identifying the subject comprising a splicing defect in an autism spectrum disorder (ASD)-associated gene, the target gene being characterized as having an hnRNP L binding site. The subject is treated by administering a spliceopathy rescue agent to repair the splicing defect. Also disclosed are methods of upregulating hnRNP L and hnRNP L targets by administering a therapeutically effective amount of an isoprenoid antibiotic. Methods of screening compounds for use in treating autism spectrum disorder (ASD) are also described.
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