Scientists have successfully edited the DNA of the mice with specific gene mutation which in turn will prevent them from going completely deaf.
According to the study published in the journal Nature, the gene-editing tool CRISPR-Cas9 was injected in the ears of the mice by the researchers with a deafness-causing genetic mutation. They have disrupted a gene variant using molecular scissors which were able to cut the disease-causing copy of the gene precisely. Fyodor Urnov, the associate director at Altius Institute for Biomedical Sciences, said, “We have entered the age where the human genome is a real drug target” and also the researchers “have provided the first important step and a strong perspective of hope for people who have this mutation.”
These results were reported on Wednesday in the journal, and a genetic engineer at Broad Institute, the Massachusetts Institute of Technology and Harvard University has said, “We’re hopeful that our results will help guide the development of such strategies.” The experiment has been performed on ‘Beethoven mouse’ as these mice carry a defect that causes them to start losing their hearing at an early stage of life.
David says, “Humans that are born with even one bad copy of this gene experience progressive hearing loss that’s evident in their early childhood and by the time they reach late childhood they’re profoundly deaf.” David and his colleagues have used the genome-editing technique CRISPR-Cas9 with a lipid droplet which allows the genome editing tool to enter the hair cells.
The untreated ears were able to pick up noises that were 80 decibels or louder, after four weeks instead the injected listeners were able to hear 60 to 65 decibels.
After eight weeks, the hair cells of treated ears resembled those in healthy animals, and untreated ears looked damaged and sparse. Fyodor says, “We’re entering a time when we can genetically manipulate organs in vivo rather than manipulate cells in a dish and then transplant them. Fyodor continued saying that is remarkable and that this is happening in our lifetime.