Antisense therapy restores fragile X protein production in human cells

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.

from Latest Science News -- ScienceDaily https://ift.tt/Re8jLr9