The 2012 Deutsch Grant for Canavan Research will proudly fund Dr. Guangping Gao as he works to develop a way to administer gene therapy through an intravenous injection rather than through extensive and risky brain surgery. This grant will allow Dr. Gao to research the following questions to solidify his study:

1. Out of three leading vectors we will be testing for gene therapy, which one will be the best performer?
2. What is the minimal dose of gene therapeutics that is both safe and effective?
3. How late in the pathogenic development can we still provide treatment that will be effective?
4. How does this novel gene therapeutics work? Preventing the disease progression andor repairing the damages made to the bran and reversing the disease progression?

All of Dr. Gao’s research will be made available to the Canavan research community as well as Canavan families. Prior to the Deutsch Grant funding, Dr. Gao had narrowed down which vectors cross the blood-brain-barrier and deliver the genes to the brain and spinal cord tissues after injection. This grant will allow Dr. Gao to continue his groundbreaking study to find the best intravenous gene therapy treatment for Canavan Disease.