Neurogenetics
genomics, ALS, anti-sense
Research aim
Given increasing possibilities in gene-based therapies for ALS, the overall aim of my research is to elucidate the exact genetic contribution in every patient to liability to disease.
About us
Given increasing possibilities in gene-based therapies, most notably illustrated by the positive disease modifying effects of anti-SOD1 and potentially also anti-FUS antisense therapies in ALS, the overall aim of our research is to elucidate the exact genetic contribution in every patient to liability to disease and to disease progression. These lines of research can reveal rational targets for new and rapid drug development, a process thus far that has been going on for decades with a trial-and-error approach, with only one (Riluzole) established positive result with minimal effect. We coordinate Project MinE – which started in 2015-, an international collaboration between 27 centers in 21 countries, having sequenced now over 10,000 samples. The project has shown how to best disentangle the genetic structure of ALS in search for novel causal genes and disease modifying targets.