Much of the research into a cure for retinitis pigmentosa is genotype specific. When Usher 1F Collaborative was founded in December 2013, there was minimal research for type 1F. Today, we have provided significant funding to two major research labs that specialize in Usher Syndrome research. At one lab, significant progress has been made toward creating an animal model of the Usher 1F mutation with plans and a process in place to use this model to test a type of gene therapy. A second lab has achieved major progress toward achieving both gene replacement therapy and stem cell therapy using retinal cells made from patients’ own skin cells. With continued funding, both of these paths have the potential to result in a clinical trial in two to five years.
Right now, parents of children with type 1F are sitting and watching their children's vision fade while clinical trials for other Usher genotypes are offering encouragement and hope to others. Please help make hope a reality for those with type 1F.
"Fear, panic and down right anger has overtaken our family. As if being deaf and overcoming that obstacle was not enough, we have learned that our son will gradually lose his vision. This is a cruel, unfair disease. We need to find a cure. I will stop at nothing as his mother to raise awareness about Usher Syndrome type 1F."
"He cannot ride a bike because he can’t see other bicyclists or cars until they are right in front of him, when there’s not enough time to stop. He is learning to use a white cane, but like other teenagers, doesn’t like to be seen as different or disabled. His biggest wish is a 'fix' for his vision loss like he has for his deafness."
"With late walking and a bit of poor balance as well as the fact that Harry is of Jewish descent, we pushed for the test for Usher Syndrome in 2013 which resulted in a positive test result in February 2014 for Usher Syndrome type 1F. We are all here and determined to do ourselves out of a job in fundraising and research into a cure for Retinitis Pigmentosa and Usher Syndrome."
"I have so many dreams for the near future to be able to see while traveling the world, seeing and cooking my fabulous foods, being an endurance runner/hiker and seeing my future grandchildren with whatever vision I have left. Yet, I am so hopeful for a miracle treatment or a cure for Usher 1F."