SURF1 LEIGH SYNDROME
HELP US REACH OUR
$4.5M GOAL!
Your help is crucial in raising funds and awareness for our children. Every donation, large and small, gets us closer to a cure.
Only $3M is needed to reach our goal of a Formal Toxicology Study, GMP Drug Manufacturing, and OUR ULTIMATE GOAL OF A CLINICAL TRIAL!
Time is not on our side. Leigh syndrome is a progressive neurological disease that slowly robs children of their abilities, and eventually their lives. The sooner we reach our $3M goal the faster we get to a clinical trial. Doctors at UT Southwestern are working as quickly as possible, but funding is needed for research to progress.
Thank you for your HELP!!
Gene therapy is a revolutionary approach to treating genetic diseases, where a one-time treatment can provide a lifetime of benefits. Instead of treating just the symptoms, gene therapy fixes the disease at the source by replacing the defective gene with a healthy copy.
A healthy copy of the patient's defective gene is loaded into a virus that has been stripped of its own DNA.
Trillions of viruses, each containing a healthy copy of the gene, are injected into the patient's spinal fluid.
The viruses bind to cells in the patient's spinal cord and brain and deliver healthy gene's to the cell's nucleus.
how it works
In the case that a gene changes—also known as mutating—in a way that causes Leigh syndrome,
gene therapy may be able to help. Gene therapy is the introduction, removal or change in genetic material—specifically DNA or RNA—into the cells of a patient to treat a specific disease. The transferred genetic material changes how a protein—or group of proteins—is produced by the cell.