Children with a genetic developmental disorder often require lifelong care, which has a major impact on the lives of patients, loved ones and society. We try to find a solution by searching for the genetic cause and then repairing the defect (or mutation) using so-called ‘antisense oligonucleotides’ (ASOs).
Developing a platform
Our goal is to develop a platform for identifying successful treatment of developmental disorders. We will do this by measuring iPSCs from patients and comparing them to the control group. The iPSCs are human brain cells with the same genetic make-up as the patient’s, made from blood cells of the patient himself. We will also explore the ethical issues associated with the technology.
Ultimately, our desire is to deploy our platform to achieve successful therapy.