Novel genome editing approach restores hearing in adult preclinical models with genetic deafness.

A recent study published in Science Translational Medicine has shown promising results in using a novel genome editing approach to restore hearing in adult mice with genetic deafness. The researchers targeted a specific mutation in the microRNA-96 (MiR-96) gene that causes progressive hearing loss in mice and humans. By using a CRISPR/Cas9 genome editing approach delivered through an adeno-associated virus (AAV), the researchers were able to disrupt the mutation and preserve auditory function in the mice. The study also found that the AAV-mediated genome editing approach had a good safety profile. This research offers hope for future clinical trials aimed at restoring auditory function in people with genetic forms of hearing impairment. [Extracted from the article]