Genetic neurological disorders are challenging to treat, let alone cure due to the complexity of the brain and the barrier imposed by the blood brain barrier. Our research team are testing gene therapies, including gene editing technologies such as CRISPR gene editing tools to create novel therapies which have the capacity to cross the blood brain barrier and permanently correct mutated genes back to wild type in neuronal cells.To test this our lab uses mouse and cellular models including 2D neuronal cultures and 3D brain organoids.
