Developing treatments for genetic diseases and cancers caused by nonsense mutations
Monogenic diseases are all rare diseases but, collectively, they
affect 300 million people worldwide.
More than 7,000 monogenic diseases have already been described.
In each case, a single gene defect impairs in various ways the production of the corresponding protein in its fully functional state, causing the disease.
The therapeutic need is huge but there are at present almost no curative treatments.