Gaucher's disease, one of the so-called "rare diseases", affects approximately 30,000 people worldwide. This hereditary disease causes the accumulation of fatty substances in certain organs, which in turn produces abnormalities, some of which can prove fatal. Andrés Klein, using a strategy that identifies the genes which modify the progression of Gaucher's disease, has discovered a way of predicting what type of disease the patient will develop.
Klein's work could be the first step towards creating personalized therapies in the future for every subtype of patients with this disease, which would have a huge impact on their quality of life. In addition, the strategy developed by this pioneer could be applied to other types of diseases like Parkinson's.
Klein has been endorsed by several institutions including CONICYT, the Mount Sinai School of Medicine, the Pew Charitable Trusts in the USA, the Ara Parseghian Medical Research Foundation in Israel and the Gaucher Association in the United Kingdom.