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 several rare genetic diseases such as SURF1 Leigh syndrome. We are fortunate to partner with the team at UTSW who have years of experience in this field and are at the forefront of gene therapy research. Our hope is that this research project will lead to an FDA approved clinical trial, which would be the first step in finding a cure for this devastating disease.
The pediatric neurology team at UTSW is dedicated to finding treatments for a range of neurodegenerative conditions, and this project will be an important step in finding a viable treatment for many other diseases related to genetic and mitochondrial defects.