A research project funded by FRAXA has led to new fragile x gene therapy initiative, this time in France.
Lysogene, a French biopharmaceutical company working to develop gene therapy treatments for brain disorders, is partnering with FRAXA Investigator Dr. Herve Moine to tackle fragile X syndrome.
They aim to develop a new therapeutic approach based on the pioneering work of Dr. Hervé Moine, which showed that the fragile X protein (FMRP) regulates production of the critical enzyme DgkK, and an absence of FMRP results in reduced DgkK. Lysogene is using gene therapy to increase DgkK.
With a 2015-2016 $90,000 grant from FRAXA Research Foundation, Dr. Herve Moine and colleagues successfully tested this approach (boosting DgkK) in a mouse model of fragile X syndrome. There is only so much that a small molecule (drug) can do to increase levels of a deficient protein; however, gene therapy can directly promote synthesis of new protein, correcting the deficit.