Ontology highlight
ABSTRACT:
SUBMITTER: Gurney ME
PROVIDER: S-EPMC5677090 | biostudies-other | 2017 Nov
REPOSITORIES: biostudies-other
Gurney Mark E ME Cogram Patricia P Deacon Robert M RM Rex Christopher C Tranfaglia Michael M
Scientific reports 20171107 1
Fragile-X syndrome (FXS) patients display intellectual disability and autism spectrum disorder due to silencing of the X-linked, fragile-X mental retardation-1 (FMR1) gene. Dysregulation of cAMP metabolism is a consistent finding in patients and in the mouse and fly FXS models. We therefore explored if BPN14770, a prototypic phosphodiesterase-4D negative allosteric modulator (PDE4D-NAM) in early human clinical trials, might provide therapeutic benefit in the mouse FXS model. Daily treatment of a ...[more]