TARGETED TREATMENT OF AUTISM SPECTRUM DISORDER AND OTHER NEUROLOGICAL OR PSYCHIATRIC DISORDERS

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.