NEW ORLEANS (AP) - In a promising step against a genetic disease that causes deafness and gradual loss of vision, scientists have partly restored hearing with a single injection to young mice.

Experts praised the study on Usher syndrome, published online last week by the journal Nature Medicine, but the results are still a long way from preventing the disease.

Children with Usher _ an estimated one in 6,000 to 7,500 babies worldwide _ are born deaf. The visual component of the disease, called retinitis pigmentosa, often starts in childhood, but severe problems are more likely in adolescence and young adulthood. About half of adults with Usher can still read a newspaper into their 50s.

Some groups, such as Louisianas Cajuns and Canadas Acadians, have an unusually high prevalence of the disease.

In the study, Michelle Hastings of Chicago Medical School and Jennifer Lentz of the Louisiana State University Health Science Center found that one injection allowed affected mice to hear for months.

The injection, or patch, is a laboratory-created fragment of RNA, which is a chemical cousin of DNA. The patch was designed to interfere with the effects of a faulty gene.

Its a very promising and striking finding, said Dr. Henry Paulson, a University of Michigan specialist in neurodegenerative diseases.

Curing a mouse is quite different from curing a human, he said, but hes cautiously optimistic.

Lentz, the studys lead author, and Hastings, senior author, have received a $1.3 million grant from the National Institutes of Health for four more years of mouse studies to learn the treatments physical effects and to refine timing of shots.

Time erodes the effects of the gene-inhibiting patch over the faulty RNA, and researchers plan to test whether booster shots retain hearing in treated mice. Theyre also beginning work to see whether the treatment prevents gradual loss of vision.

Read more:
Partial fix found in mice for genetic disease

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