Our mission is to fund SURF1 gene therapy research at UT Southwestern Medical Center in Dallas, Texas. The field of gene therapy has gone through remarkable breakthroughs these last few years and offers incredible promise for genetic diseases such as SURF1 Leigh syndrome.
help us reach our goal!
Your help is crucial in raising funds and awareness for our children. Every donation, large and small, gets us closer to a cure. $3.3 million is needed to reach our goal, which is an FDA approved clinical trial for a gene therapy treatment for Surf1 Leigh Syndrome.
Time is not on our side. Leigh Syndrome is a progressive disease that slowly robs children of their abilities, and eventually their lives. The course of the disease varies greatly with each child, but the median lifespan is just 2.5 years. Doctors at UT Southwestern are working as quickly as possible, but funding is needed for research to progress.
Pre-clinical (test safety/efficacy in mouse model), cost $300k, FULLY FUNDED!
Formal toxicology study, cost $350k, FULLY FUNDED!
Raw materials and prep work for AAV/SURF1 vector, cost $800k, NEED TO START ASAP.
Manufacture the clinical AAV/SURF1 vector, cost $800k.
Conduct the FDA approved human clinical trial, cost $1M.
*donation total updated on 10/05/19*