Statins for fragile X

Researchers at the Massachusetts Institute of Technology have mouse data showing that the cholesterol drug lovastatin can correct fragile X syndrome.1 The findings add to a growing list of unconventional strategies for treating this common form of mental retardation.

Fragile X syndrome is caused by trinucleotide repeat expansions in fragile X mental retardation 1 (FMR1). The condition causes alterations in protein synthesis in neurons, leading to developmental delays, autism and epilepsy.

FMR1's normal function is to negatively regulate protein synthesis. Indeed, Fmr1 knockout mice generally exhibit increased protein levels. Thus, therapeutic strategies for the condition have aimed to lower overall rates of protein synthesis by hitting translation factors and brain receptors that regulate translation.2


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