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Antisense therapy restores fragile X protein production in human cells

July 5, 2023
This post was originally published on this site

An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome. This breakthrough was possible because of the novel findings, also presented in the study, that aberrant alternative splicing of messenger RNA (mRNA) plays a principal role in fragile X syndrome, the most common form of inherited intellectual disability and the most frequent single-gene cause of autism.