A Huge Step for Gene Therapy
A new gene therapy from researchers at National Taiwan University Hospital gives hope to children born with an extremely rare brain disorder that blocks communication between cells in the nervous system. Known as Aromatic L-amino acid decarboxylase (or AADC) deficiency, this debilitating disorder makes even easy tasks, like eating, impossible without help.
Dr. Hwu Wuh-Liang and his team were able to help one girl with the disease – six-year-old, Chichi – by injecting a new gene therapy drug into the part of the brain that controls movement. Now, Chichi is showing signs of significant improvement.
Ultimately, this discovery could lead to more advanced gene therapy solutions for other brain disorders, as well.
Like Dr. Hwu Wuh-Liang says, “I think this is not the ultimate cure, but we allowed her to survive and she began to grow and develop. In the future, she will be able to receive newer and better treatments. I think it brings hope to the entire field [of gene therapy.”