Gene therapy restored hearing in a mouse model of human hereditary deafness, reports a team led by researchers at the Louisiana State University Health Sciences Center, in New Orleans, and the Rosalind Franklin University of Medicine and Science, in North Chicago, Ill (Lentz JJ et al. Nature Med. doi: 10.1038/nm.3106 [published online February 4, 2013]).
The researchers used a mouse model of Usher syndrome, the leading genetic cause of combined deafness and blindness. This condition is often associated with mutations in the USH1C gene that lead to abnormal splicing of RNA (the process that removes the parts of a gene that do not code for amino acids) and to the production of a truncated version of the protein harmonin.
Hampton T. Gene Therapy to Restore Hearing. JAMA. 2013;309(9):865. doi:10.1001/jama.2013.1844