The gene repair therapy project is continuing, albeit slowly. A young man's death after gene therapy was widely reported in the news. This recent tragedy raised serious questions about gene therapy. The good news is that the gene repair therapy by Kimeragen does not use viruses as carriers for the gene. Even the modified viruses used in most gene therapy can cause inflammatory responses. The gene repair that is the hope for a cure in MSUD uses a molecule called a chimeraplast - a combination of DNA and RNA sequences that direct the body's own molecular tools to repair the gene. It differs from regular gene therapy and is expected to reduce the risks.
Human trials on children with Crigler-Najjar Disease are planned for sometime next year. Extensive testing is being done to insure the safety of the therapy.
- from a conversation with Dr. Michael Blaese