In the fall of 1994, Drs. Roger and Helene Karlin met with Dr. Matthew During and Dr. Paola Leone at Yale University and inspired them to attempt to develop a gene therapy approach. Within a year and a half, they had developed a vector system to transport the new genes into brain cells. These new genes were then administered into the ventricles (the area between the brain where spinal fluid flows) of Lindsay and another affected child, Alyssa Mushin.

The gene therapy was performed in Auckland, New Zealand on March 6, 1996. It was the world's first gene therapy for a genetic brain disease. Both children showed remarkable improvements, such as increased alertness, improved vision and stronger head control. They had awakened to the world.

News of the research spread quickly: it was featured on "Sixty Minutes" and "The CBS Morning Show" as well as in The New York Times, TIME Magazine, and on the cover of The South China Morning Post (a prominent Asian newspaper), opposite Bob Dole's bid for the presidency. As more families learned of the research, they, too, joined the cause. The Canavan Research Foundation took on the task of raising funds, of tackling regulatory roadblocks, and of being the champions for their children. After a year and a half of various delays, the same gene therapy, slightly modified, was approved by the FDA in January of 1998 to be used in a second experimental trial at Yale University and Jefferson Medical College. Sixteen children ranging in age from 9 months to 7 years were treated in the first clinical trial for a genetic brain disease in the United States All of the children showed improvement, and the results have been published in various scientific journals. Meanwhile, the research for Canavan Disease was recognized in the Human Genome Exhibit at the Museum of Natural History in New York for its role in advancing gene therapy to cure disease.