GENETIC ENGINEERING: GENETIC DISEASES

The Fragile X Syndrome

Substitute for the function of the protein

If we knew precisely what the fragile X protein does in nerve cells,
we might find ways to bypass the need for it. We now know that FMRP
aids in the production of certain proteins some of which appear to be
necessary for communication at the synapses - the connections between
the brain's nerve cells. An exciting current research direction is
identifying the mRNAs that are either overexpressed or underexpressed
when FMRP is lacking. Since mRNAs code for proteins, this may tell us
which proteins depend on FMRP for their normal expression in cells.