BOSTON -- In a laboratory at Boston Children's Hospital a cure for genetic deafness is taking shape. Lead researcher Jeff Holt says that if all goes as planned, children of the future who lose their ability to hear due to genetic mutation will never go DEAF.
Holt and his fellow researchers are attacking the problem at its source. They are using engineered viruses to repair damaged genes that make up parts of the inner ear.
"Our strategy was to take a viral vector, remove the viral genes so that it doesn't make anyone sick and to replace those with the correct DNA sequence for TMC1," said Holt, an associate professor of Otolaryngology.
TMC1 is a gene critical to hearing. It is responsible for encoding proteins that convert sound into electrical signals the brain can process.
To test their treatment protocol, Holt and his team used two types of Deaf mice that model the dominant and recessive genetic mutations of TMC1 in humans. The team used an engineered virus called adeno-associated virus, or AAV1, to deliver the functioning TMC1 gene to the inner ears of the Deaf mice models...
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