Self-Regenerating Retina One of Six New FFB-Funded Research Efforts
An emerging therapy to restore vision caused by retinal diseases—or potentially traumatic injury—is one of six new research projects being funded by the Foundation Fighting Blindness. The research effort is being led by Tom Reh, Ph.D., at the University of Washington. He’s investigating how Muller glia—cells that provide the retina with structural support—can be manipulated to also sprout new photoreceptors, the cells in the retina that process light and enable people to see.
A total of $1.5 million in funding is being allocated for the six new research efforts, which cover a broad range of emerging therapeutic approaches. The projects were selected during the annual grants review process conducted by the Foundation’s Scientific Advisory Board, when it convened at VISIONS 2014, the Foundation’s annual conference, in Colorado in June.
“We are very excited about the sight-saving potential of these projects for a variety of people with retinal diseases,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “There’s a lot of great innovation in these new efforts, but equally important, their scientific methodology is strong. That’s critical for success.”
Seventy-five projects were originally submitted for consideration last fall by members of the international retinal research community. Twenty-five of the most promising proposals made the first cut. Ultimately, funding was available for the top six.
Here are summaries of the five other new projects:
Suppressing thyroid hormone to save cones
Xi-Qin Ding, Ph.D., at the University of Oklahoma, is continuing lab studies which have demonstrated that suppression of the thyroid hormone can be a viable approach for preserving cones, the cells that provide central vision and the ability to perceive detail, in people with retinal diseases. One key to the success of this potential treatment is localizing it to cones, perhaps by delivering it as an eye drop. Localization to the retina is important because the thyroid hormone is critical to the rest of the body’s development and metabolism.
Identifying vision-saving drugs
James Fadool, M.D., is screening thousands of potential vision-saving drugs in zebrafish models of retinal diseases. An advantage of these neuroprotective therapy candidates is that they have the potential to work for many eye diseases, independent of the vision-robbing genetic mutation.
Gene therapy for autosomal dominant RP
Alfred Lewin, Ph.D., at the University of Florida, is developing a two-step gene therapy for autosomal dominant retinitis pigmentosa (adRP) caused by mutations in the gene rhodopsin. RP often has an autosomal dominant inheritance pattern, meaning one copy of an altered gene in each cell can cause the disorder. People with adRP have an affected parent and often other family members with the disorder. Dr. Lewin’s treatment involves delivery of a healthy gene and suppression of the mutant gene. Success in the studies will help position the treatment for evaluation in a clinical trial.
Delivering the large Usher syndrome 1B gene
Shannon Boye, Ph.D., at the University of Florida, is developing a gene therapy based on an adeno-associated virus for Usher syndrome type 1B, which is caused by mutations in the gene MYO7A. Because MYO7A is large, the therapy involves delivery of the gene in two packages— an approach known as dual-vector delivery. Dr. Boye’s emerging treatment provides an alternative to UshStat®, a gene therapy for Usher syndrome type 1B currently in a human study. UshStat uses a high-capacity, single-vector lentivirus for gene delivery.
Fixing errors in genetic code processing
Jonathan Staley, Ph.D., at the University of Chicago, is investigating how alterations in splicing-factor genes—genes which ultimately help retinal cells properly process genetic code—result in retinal diseases, such as RP. He and his research team are developing strategies for correcting these alterations and hopefully saving vision.