One Shot

The science of hope, delivered in a single dose.

Children with SURF1 Leigh syndrome are fighting a rare, fatal genetic disease. A promising gene therapy is already developed and could reach clinical trial with your help.

This one-time treatment has the potential to change — and save — lives. Help us fund their one shot.

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Contribute directly to the SURF1 clinical trial and help drive progress forward. Bring gene therapy to those who are waiting.

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What exactly is SURF1 gene therapy?

It is a “gene replacement” treatment for Leigh Syndrome, a rare brain disorder. The treatment uses a tiny, harmless delivery vehicle to carry a healthy copy of the SURF1 gene into the body’s cells. This healthy gene helps the body produce energy properly, which is something children with SURF1 mutations struggle to do.

How is the treatment given?

It is usually delivered through a single injection into the spinal fluid (intrathecally). This helps the healthy gene reach the brain and spinal cord, where it is needed most.

Will it cure the disease completely?

While scientists hope it will be a permanent fix, the main goal is to stop the disease from getting worse and help the body create more energy. Preclinical studies have shown that it can improve muscle strength and reduce harmful acid buildup in the blood.

Why is SURF1 gene therapy so expensive?

SURF1 gene therapy is expensive because it targets an extremely rare disease, meaning the massive costs of research and clinical trials are shared by very few patients. Developing a treatment that can safely reach the brain and spinal cord is technically difficult and requires specialized, high-tech labs rather than standard factories. While the multi-million dollar price tag is high, it is designed as a one-time fix to replace a lifetime of constant hospital visits and specialized medical care.

Is gene therapy the future of medicine?

Many researchers believe gene therapy could play a major role in the future of healthcare, and it is often referred to as the “third pillar” of medicine. While traditional medicine treats symptoms with daily pills, gene therapy aims to provide permanent, one-time cures by fixing the root genetic cause of a disease. As technology advances, it is expected to move beyond rare diseases to help treat common conditions like heart disease, cancer, and chronic pain.

Will it eventually replace traditional drugs?

It won’t replace everything, but it will transform how we handle “incurable” conditions. By shifting healthcare from lifelong management to single-administration cures, it has the potential to save the medical system money over time and significantly improve the quality of life for millions of patients.

These are some of the children at the heart of this mission. Visit the fundraisers on our leaderboard to learn more about their stories and why this fight matters so deeply.