Retinal dystrophies are hereditary diseases that alters the anatomy or function of the retina, causes severe vision loss, and are passed down from generation to generation. Currently, there is no cure, but cell and gene therapies continue to show promise as possible treatment options.
In Europe, a group has artificially created photoreceptor precursor cells outside the body using stem cells. Photoreceptor precursor cells are cells that eventually turn into cones or rods. They then transplanted these cells into a mouse with autosomal recessive retinitis pigmentosa to see what happens.
In mice with end-stage retinal degeneration, only 1.5% of the precursor cells actually integrated into the retina. However, 33% of these successful integrations responded to light, and 50% showed visual behavior. While more work needs to be done before this is ready for the clinic, this may turn into a potential therapy option for those with end-stage retinal degeneration.
What this means for Usher Syndrome: If proven successful, Usher patients with advanced retinal degeneration may one day be able to regain some basic visual function.