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Gene Therapy

Gene Therapy Research at Nationwide Children's

The TBCD fundraiser Miracle for Max has agreed to finance funding to The TBCD Project to fund the development of an organic model for TBCD research at Bradbury Lab at Nationwide Children's Hospital. 

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. Your child will be seen at their Leukodystrophy clinic, where your child's samples will be collected to build a firmer base of research upon, and the hospital will have your history and contact information when it's time for clinical trials.

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 donating to The TBCD Project to help us 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.