Rett syndrome currently has no cure. Now, innovations in gene therapy bring the possibility of treating the root cause of the disease closer than ever before.
The therapy met its primary endpoints in two Phase 3 studies and offers a new approach for the tens of millions of people living with the psychiatric condition.
By using extracellular vesicles to target specific neurons in the hypothalamus, scientists induced weight loss in a mouse model of obesity without altering its diet or exercise.