18.08.2020RNA as a future cure for hereditary diseases

ETH Zurich scientists have developed an RNA molecule that can be used in bone marrow cells to correct genetic errors that affect protein production. Patients suffering from a rare hereditary disease that causes a painful hypersensitivity to sunlight could benefit in future.

Short RNA molecules can be used as medication. Their effectiveness is based on the genetic information they carry: therapeutic RNA can bind to the body’s own RNA and thus influence how it functions. However, only a handful of such drugs are available so far.

“That’s mainly because it’s tricky to get the RNA molecules to precisely the organ in the body where they need to take effect. Currently, that’s the biggest hurdle in the development of RNA drugs,” says Jonathan Hall, Professor of Pharmaceutical Chemistry at ETH Zurich. Together with Daniel Schümperli, Emeritus Professor from the University of Berne, and colleagues from ETH, University Hospital Zurich and Triemli Hospital Zurich, he has now succeeded in developing an RNA molecule that can compensate for the effect of gene mutations in bone marrow cells.

This therapeutic approach could one day be applied to a rare hereditary disease called erythropoietic protoporphyria (EPP). Those who suffer from EPP experience a painful sensitivity to sunlight.