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Gene Therapy

Gene Therapy Research at Nationwide Children's

Currently, early proof of concept studies are underway to understand the disease, develop model systems, and evaluate potential genetic therapies. No treatment studies or evaluations are being done on any patients at this time, but we are hopeful for the future. Genetic reports and tissue samples are highly welcomed and needed to further this research.

What is Gene Therapy?

Gene therapy is a cutting-edge new treatment method that completely replaces a defective gene with a working one in a patient's genetic code. Unlike drugs or treatments that are often ongoing for life, gene therapy corrects the underlying problems in the genetic code itself.

How Can I Help?

The more TBCD patients that can be seen at Nationwide Children's Hospital, the stronger the data will be. 

Visit Nationwide Children's

Visit Nationwide Children's

Consider joining our cohort at Nationwide Children's Hospital. Patients will be seen at their Leukodystrophy clinic, where their samples will be collected to build a firmer base of research upon. This also will help them identify potential candidates for clinical trials that may take place as a result in the future. There are currently no clinical trials taking place or slated to take place. Please do not contact the hospital asking to register for a trial

Help Us Find Grants

Help Us Find Grants

While the first model is already being funded, future phases are astronomically expensive. The estimated cost of research and development to clinicals is $2m. Our families cannot cover that cost. Grant funding will be instrumental in being able to pay for this work. Any research toward potentially applicable grants is extremely helpful.

Finance Research

Finance Research

Please consider making a tax-deductible donation to Bradbury Lab to help cover the costs of AAV gene therapy development. TBCD is a disease with a very small patient population, which means we desperately need outside help to be able to achieve our funding goals.