Harrison Wren GABEL,Benyam KINDE,Michael E. GREENBERG
申请号：
US15556665
公开号：
US20180318285A1
申请日:
2016.03.09
申请国别(地区):
US
年份:
2018
代理人:
摘要：
We have determined that MeCP2 protein mediates modulation of long-gene expression in the brain and results in neurological dysfunction associated with autism spectrum disorders, including but not limited to, Fragile X Syndrome, Rett syndrome, and Angelman syndrome (AS). In particular, a lack of MeCP2 protein causes up-regulation of long gene expression in the brain which corresponds with the pathology of Rett syndrome and Fragile X Syndrome, while too much MeCP2 protein results in excessive repression of long gene expression in the brain and pathology related to MeCP2 duplication syndrome. Accordingly, embodiments of the invention are directed to methods for treatment of autism spectrum disorders. The methods involve administration, to a subject, agents that modulate the expression of long genes in the brain.
