Fragile X syndrome （FXS） is the most common cause of inherited intellectual disability and the most common known genetic cause of autism or autism spectrum disorders. FXS is caused by silencing or mutation of the fr...Fragile X syndrome （FXS） is the most common cause of inherited intellectual disability and the most common known genetic cause of autism or autism spectrum disorders. FXS is caused by silencing or mutation of the fragile X mental retardation gene （FMR1）, a known RNA-binding protein that acts as a negative regulator of translation [1, 2]. FXS patients demonstrate a myriad of symptoms that can vary widely between individuals, including impaired cognition, physical abnormalities, sleep problems, hyperarousal to sensory stimuli, increased anxiety, obsessive compulsive disorder-like behavior, attention-deficit hyperactive disorder symptoms, self-injurious behavior, aggression, and increased risk of seizures [3]. The molecular mechanisms underlying FXS are not clear, and currently there is no ideal treatment.展开更多
基金supported by grants from the National Natural Science Foundation of China(31471125 and 31671215)the‘‘One Thousand Talents Plan of China’’
文摘Fragile X syndrome （FXS） is the most common cause of inherited intellectual disability and the most common known genetic cause of autism or autism spectrum disorders. FXS is caused by silencing or mutation of the fragile X mental retardation gene （FMR1）, a known RNA-binding protein that acts as a negative regulator of translation [1, 2]. FXS patients demonstrate a myriad of symptoms that can vary widely between individuals, including impaired cognition, physical abnormalities, sleep problems, hyperarousal to sensory stimuli, increased anxiety, obsessive compulsive disorder-like behavior, attention-deficit hyperactive disorder symptoms, self-injurious behavior, aggression, and increased risk of seizures [3]. The molecular mechanisms underlying FXS are not clear, and currently there is no ideal treatment.
