Molecular therapy improves hearing and balance in a mouse model of a hereditary disease that causes deafness and balance disorders.

In humans, Usher syndrome is often caused by a mutation in the gene USH1C that results in the production of a truncated form of the protein harmonin — which normally guides development of the inner ear (pictured) — and leads to impaired hearing.

Credit: JENNIFER LENTZ

Jennifer Lentz at Louisiana State University in New Orleans, Michelle Hastings at Rosalind Franklin University in North Chicago, Illinois, and their collaborators injected newborn mice with DNA-like molecules that mask the mutation in the cell's protein-expression machinery, causing normal harmonin to be produced. This stopped the loss of inner-ear cells that respond to sound, improved low- and mid-frequency hearing and reduced behaviours associated with balance impairment such as head-tossing and circling. The effects lasted at least six months. The authors suggest that a similar approach may have therapeutic potential in human congenital deafness.

Nature Med. http://dx.doi.org/10.1038/nm.3106 (2013)