Researchers at Harvard Medical School and Boston Children’s Hospital have found a potential treatment for hereditary deafness.
Beethoven’s 5th symphony is a cornerstone of classical music. Hard to believe the composer was almost completely deaf from a genetic condition when he finished it.
David Corey, Ph.D., Prof. of Neurobiology Harvard Medical School said, “These children are born fairly normal, but then over ten or 20 years, they lose their hearing.”
Now, these aptly named Beethoven mice might hold the key to a potential cure. Scientists believe the animals have a defect in the same gene that most may have caused Beethoven’s deafness.
“Our genome is composed of about three billion letters of DNA that together make up 20,000 genes. For the disease we’re studying, one mistake in the DNA in one of the genes causes deafness,” said Doctor Corey.
Researchers identified that as a hearing gene called TMC-1. It’s a gene that comes in pairs.
Using a newly-refined gene-editing system, they disabled the defective copy of the TMC-1 gene, leaving the good gene in place.
“By eliminating just the bad copy, that would be sufficient to preserve hearing,” said Doctor Corey.
The scientists then delivered the edited DNA back into the cells of the mice and tested their hearing.
Jeffrey Holt, Ph.D., Prof. of Otolaryngology Boston Children’s Hospital said, “We put little scalp electrodes on the back of the head, play sounds into the ear, and can measure the brain activity in response.”
Researchers say the mice were able to hear sounds as low as 45 decibels, the level of a quiet conversation.
“This could be life-changing a famed composer, his namesake mice, and a team of scientists using cutting-edge medicine to help people who would otherwise go deaf, Hear,” said Doctor Holt.
The scientists say this research paves the way for using the new editing system to treat as many as 3,500 other genetic diseases that are caused by one defective copy of a gene.
It’s important to note that Jeffrey holt holds patents on TMC-1 gene therapy.