New drug target identified for Fragile X syndrome

UCLA Health researchers have identified a potential drug target for treating Fragile X syndrome, the most common genetic ...
The New England Journal of Medicine

The Spectrum of Fragile X Disorders

Mutations in FMR1, the gene encoding fragile X messenger ribonucleoprotein 1 (FMRP), located at Xq27.3 and discovered in 1991, are the leading single-gene causes of autism and intellectual disability.
The Conversation

Fragile X syndrome often results from improperly processed genetic material – correctly cutting RNA offers a potential treatment

For many people with fragile X, the mutated gene that causes symptoms is active rather than silenced. Thom Leach/Science Photo Library Fragile X syndrome is a genetic disorder caused by a mutation in ...