GENETIC ENGINEERING: GENETIC DISEASES

The Fragile X Syndrome

Make and deliver the protein by some other means

Cells use a signaling pathway (or molecular cascade) to trigger the
synthesis of FMRP. If we understood how FMRP is normally made and
where it needs to be made, we might find a way to bypass complications
in this pathway caused by the fragile X mutation, such as impaired
translation into protein. Knowledge of this pathway could also provide
alternatives to bring about the expression of other proteins regulated
by FMRP.