The Battle Against Our Own Genes

We have seen remarkable progress in treating, and even curing, severe genetic disorders such as sickle cell disease. But these therapies cost millions and with over 4,500 rare genetic disorders to treat, it's been estimated that this will take over 2,000 years and cost more than the USA’s whole economy. Fortunately, these therapies have a superpower – developing one makes the next one much easier to develop. To harness this superpower, a coordinated effort is underway across many scientists, clinicians, and agencies with the hope of transforming the lives of those affected by these disorders.

The brain has lots of secrets – there’s still so much to discover! I am passionate about understanding neurological diseases and finding new therapies. In the lab, I try to uncover how neurons function by looking at RNA editing, a fascinating phenomenon where molecules can modify the cells’ instruction manual after it has been printed. Through understanding this phenomenon in health and disease, and learning how to ‘correct’ the cell instructions, scientists like me will be able to find cures for genetic diseases.