Géléoc believes another significant hurdle is the cost of gene therapy. The famous Gelsinger case tainted the public’s view of the technology and raised questions concerning safety.ĭr. His immune system interpreted the virus as a foreign invader and attacked, leading to the first publicly identified death caused by gene therapy. In 1999, 18-year-old Jesse Gelsinger was injected with a vector during a gene therapy clinical trial at the University of Pennsylvania. The promise gene therapy brings is not without important obstacles. She stresses the need to conduct tests on larger animal models such as non-human primates or pigs before moving on to people. Géléoc hesitates when asked about applying her research to human clinical trials. “I really was skeptical, but we did it again and confirmed that we indeed were getting good recovery. The first time we saw this, I actually did not believe it because it was almost too beautiful to be true,” says Dr. “This was basically the auditory sensitivity a normal mouse would have. Treated mice could detect sounds as low as 30 decibels, which can be compared to a whisper. Normal mice detected sound in the 20-decibel range, while no electrical response was observed in genetically deaf mice at 110 decibel tones. To determine if treatment restored hearing, researchers measured activity in the auditory, that is, hearing related, regions of the mice brains after applying sounds of varying decibels. Researchers used a synthetic vector to deliver the corrected USH1C gene into the inner ear. Mutations in the protein harmonin prevent proper growth of hair cells and lead to genetic deafness. Outer hair cells in the inner ear first amplify sound waves, and inner hair cells use the amplified vibrations to send messages to the brain. Géléoc describes as “the nucleus of the Usher group of proteins.” Harmonin helps hair cells in the cochlea, a spiral structure in the inner ear, convert sound into electrical signals. USH1C gives instructions to make a protein called harmonin, which Dr. Géléoc and a team of researchers studied the effect of a specific gene, called the USH1C gene, in a model of Usher syndrome in mice. Without a sense of balance and the ability to see and hear, many Usher syndrome patients live in an isolated world.ĭr. Usher syndrome, the most common cause of deaf-blindness, is a genetic disorder that can cause loss of balance. Kirby Neurobiology Center at Boston Children’s Hospital has explored the use of gene therapy in treating Usher syndrome. Gwenaelle Géléoc of the Department of Otolaryngology and F.M. Now, the corrected gene can give instructions to make important proteins that keep us healthy.ĭr. A vector, which is typically a virus that’s been modified to prevent it from causing disease, acts as the “truck” to deliver a corrected gene into the target tissue. The cracked window is like a defective gene. This process is similar to how gene therapy in humans works. The new window is installed, and the window’s function is restored. So, you call a window replacement company that arrives at your house with a truck carrying materials. What’s the best way to solve this problem? A new Plexiglas® window would probably be the most effective long-term solution by ensuring a high quality, likely more resilient fix. Treated mice hear noises at a much lower decibel than untreated miceĪ baseball hits your window and leaves behind a large crack.Doctors at Boston Children’s Hospital have explored a mutation in the USH1C gene by replacing the defective gene with a functional gene in mice.Usher syndrome causes hearing loss and blindness and has no treatment.
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