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.


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. (2013)