gene editing therapy

June 3, 2021

Patient of Monroe Carell Jr. Children’s Hospital at Vanderbilt first in world to receive new investigational gene editing therapy

A 9-year-old patient of Monroe Carell Jr. Children’s Hospital at Vanderbilt is the first in the world to receive an investigational gene editing therapy for Methylmalonic Acidemia (MMA), a rare genetic disorder diagnosed at birth.

By Christina Echegaray