Gene therapy provides a path to a cure for AHC.
A similar approach has already been successful in many diseases like Batten’s disease, Friedreich’s Ataxia, Huntington’s disease, Niemann-Pick disease, Parkinson’s disease, Rett Syndrome, Giant Axon Neuropathy, Multiple Sclerosis, Spinal Muscular Atrophy, and multiple retinal disorders. This is not only cutting-edge science, but also a proven, effective, and permanent fix of the genetic code.
It will be delivered by a one-time injection into the cerebrospinal fluid and is designed to permanently correct the dysfunction caused by a mutated gene. Unlike drugs, gene therapy targets the source of the problem: DNA. In many diseases it permanently corrects the dysfunction caused by a problematic gene. We call that a cure.
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We are so close to trying Gene Therapy with Dax! If you can donate directly to this cause please do.... We appreciate EVERY Dollar donated!!
With funds raised from the AHC Foundation, we will support AAV gene therapy and other research related to the project. We believe a path has been identified to a viable permanent treatment: gene therapy. The gene therapy we propose will involve inserting a functional copy of the ATP1A3 gene into a virus (an adeno-associated virus or AAV) that has been stripped of its harmful payload and its ability to reproduce. The virus will then be injected into the cerebrospinal fluid and will carry the functional gene to the cells in the brain that are failing. By delivering additional functional copies of the gene, we should be able to rescue those cells, and treat the disease permanently.