FMRP Expression Levels in Mouse Central Nervous System Neurons Determine Behavioral Phenotype.

ABSTRACT: Fragile X mental retardation protein (FMRP) is absent or highly reduced in Fragile X Syndrome, a genetic disorder causing cognitive impairment and autistic behaviors. Previous proof-of-principle studies have demonstrated that restoring FMRP in the brain using viral vectors can improve pathological abnormalities in mouse models of fragile X. However, unlike small molecule drugs where the dose can readily be adjusted during treatment, viral vector-based biological therapeutic drugs present challenges in terms of achieving optimal dosing and expression levels. The objective of this study was to investigate the consequences of expressing varying levels of FMRP selectively in neurons of Fmr1 knockout and wild-type (WT) mice. A wide range of neuronal FMRP transgene levels was achieved in individ

SUBMITTER: Arsenault J 

PROVIDER: S-EPMC5178026 | biostudies-literature | 2016 Dec

REPOSITORIES: biostudies-literature