Treatment options for rare diseases of the muscle, bone or nervous system

Treatment options for rare diseases of the muscle, bone or nervous system

The discovery of novel therapeutics for rare diseases affecting muscle, bone or nervous tissue is often limited by inadequate understanding of complex molecular pathogenesis and unclear links between genetics and phenotypes.

Expected results:

Our aim is to develop a system of models to help us better understand the genetic and phenotypic links in rare diseases affecting muscle, bone or nervous tissue, pathogenesis or underlying mechanisms of disease progression .

Solutions

We are looking to use primary cells or IPSC-derived cells in the matrix to develop a set of novel culture systems in vitro that will allow us to better understand disease pathology and identify new targets and solutions.