|By Joseph Scalise | 3 years ago|
A new gene-editing technique may be able to prevent at least one form of inherited deafness, according to new research published in the journal Nature.
In the study, researchers from the Massachusetts Institute of Technology and Harvard University conducted a series of experiments on Beethoven mice, which carry a defect that causes them to lose their hearing early in life.
To combat that hearing loss, the scientists in the study used the gene-editing technique CRISPR Cas-9 in the ears of the mice one day after they were born. Once inside the ear, the editor targeted the mutant gene responsible for the deafness and cut the DNA in a way that prevented it from poisoning and killing ear hair cells. As the mice still have a second, healthy version of the gene, the team believed eliminating the defective one would lead to better hearing.
Data showed that nearly all of the mice with gene-editing ears could hear better a month later. In fact, the treated ears could hear noises as low as quiet conversations. In contrast, un-injected ears in the same mice could not hear anything quieter than a garbage disposal.
This study follows past experiments that used a different gene-editing technique to alter the same inherited form of deafness in mice. Such methods could one day be used to prevent hearing loss in babies born with similar defects, and it could also combat other inherited mutations that cause hearing loss. Such methods may also potentially restore hearing to people who lost it from loud noise or infections.
“Hitherto incurable and often even untreatable diseases are now within the scope of gene therapy,” said Simon Waddington, a reader in gene transfer technology at University College London who was not involved in the research, according to The Guardian.
While more research is needed before any human trials can be run, there is no doubt the technique has potential. Gene editing is a new field that will only grow with time. Research next plan to see how safe the new technique is over extended periods of time, and they also plan to see if it will work in humans.
“We’re hopeful that our results will help guide the development of such strategies,” said study co-author David Liu, a genetic engineer at the the Massachusetts Institute of Technology, according to NPR.