Gene-based agents for the treatment of congenital eye diseases

Pharmacologists at LMU have developed gene-based agents for the treatment of congenital eye diseases. The first of these is now undergoing a phase-I clinical trial in color-blind patients at the University Medical Center in Tübingen.

Is this approach translatable in principle to other visual disorders?

Michalakis: About 200 genes have been identified which, when mutated, lead to monogenetic visual diseases, and doubtless many more remain to be discovered. Defects in any of 50 different genes can result in retinitis pigmentosa, a degenerative disease in which specific retinal photoreceptors die. Six genes have been linked to achromatopsia, and all of them should be amenable to the approach we are using to correct for defects in the CNGA3 gene.

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