FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome
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Fragile X Syndrome (FXS) is the most common inherited form of intellectual disability. It is caused by a trinucleotide expansion in the 5' UTR of the Fragile X messenger ribonucleoprotein 1 (FMR1) gene leading to loss of expression of Fragile X messenger ribo... ...
