GenSight Biologics releases additional trial data
GenSight Biologics recently published data that showed the effectiveness of GS010 – the company’s gene treatment for Leber Hereditary Optic Neuropathy, a mitochondrial disorder that can cause blindness. This therapy, like in previous studies had a bidirectional effect.
Gene Therapy
In LHON the mitochondrial protein ND4 does not express well through mitochondrial DNA. GS010, a genetherapy, causes the expression of this protein allotrope in the nucleus. It is then shuttled to mitochondria via messenger RNA. This is why GS010 can be used as a partial treatment of mitochondrial dysfunction.
Source:
https://www.leafscience.org/gensight-biologics-releases-further-trial-data/
