Foundation Receives Generous Grant from Adams Trust for Usher Syndrome Research
Research to save and restore vision for people with Usher syndrome is receiving a significant boost, thanks to a three-year, $450,000 grant awarded to the Foundation Fighting Blindness by the Marjorie C. Adams Charitable Trust. The funds will be used to support the Foundation’s diverse portfolio of Usher syndrome-related projects, including emerging gene therapies, stem-cell treatments and pharmaceuticals.
Usher syndrome is the most common cause of combined deafness and blindness, affecting more than 400,000 people worldwide. Mutations in 13 genes have been linked to the condition. Vision loss is caused by retinitis pigmentosa, a condition leading to degeneration of photoreceptors, the retinal cells that make vision possible. Hearing loss is caused by degeneration of stereocilia, cells that line the inner ear and respond to sound.
“We are delighted by the generous support from the Adams Trust, which will go a long way in supporting the Foundation’s fight against a devastating condition,” says William T. Schmidt, the Foundation’s chief executive officer. “We have a number of promising therapies in the pipeline that can benefit from this grant.”
The Foundation’s portfolio of Usher syndrome projects includes an effort led by Jennifer Lentz, Ph.D., at Louisiana State University, to develop a gene-correction therapy known as an antisense oligonucleotide. The emerging treatment, which works like a genetic patch, shows promise for saving vision in people with Usher syndrome type 1C who have Acadian (French-Canadian) heritage.
Another Usher syndrome-related project is being conducted by José Sahel, M.D., and his colleagues at the Institut de la Vision in Paris. They are working toward a clinical trial of a gene therapy which works like a drug factory, providing sustained production of a vision-saving protein called rod-derived cone viability factor, or RdCVF. The emerging treatment holds potential for saving vision in people with retinitis pigmentosa, Usher syndrome and other retinal conditions.
Also, David Gamm, M.D., Ph.D., at the University of Wisconsin-Madison, and Dennis Clegg, Ph.D., at the University of California, Santa Barbara, are developing a stem-cell patch for replacing retinal cells, and restoring vision, for people with a number of retinal diseases, including Usher syndrome.
In 2008, the Foundation received $1 million from the Adams Trust to provide six Marjorie Carr Adams Women’s Career Development Awards to women making a commitment to clinical research for retinal degenerative diseases. One recipient was Jacque Duncan, M.D., University of California, San Francisco, who now serves as chair of the Foundation’s Scientific Advisory Board.
Marjorie Carr Adams lived to 99 years old and suffered from macular degeneration. She created the trust to provide gifts that would have a meaningful impact on overcoming blindness.