Every year, there are around 4,000 babies born with genetic deafness and gene editing may be an effective method to treat them. In the human Tmc1 gene alone, there are more than 70 different mutations and researchers need to test each one to figure out exactly what they do so that we can properly restore balance and hearing in the inner ear. With this in mind, we can utilize gene therapy as a possible method to prevent deafness.
A new paper was recently published about in vivo base editing in embryonic mouse models for recessive deafness. This disorder is caused by a point mutation (one or very few nucleotides mutated in a gene in layman’s terms), in the Tmc1 (transmembrane channel-like 1) gene. The editing performed in this study was the first example of repairing a recessive gene mutation and partially restored hearing as well as hair cell sensory transduction (the ability to receive signals from the tiny hair cells that pick up sound in the ear to the brain). In case anyone is interested, here’s the link to the paper: https://stm.sciencemag.org/content/12/546/eaay9101
Stay safe, stay healthy, and be kind everyone!