Gene Therapy Milestones
- First successful gene therapy (animal model).
- Discovery of Stargardt disease gene (ABCR).
- Discovery of Usher 2A gene.
- Gene therapy restores vision in dogs with Leber congenital amaurosis.
- More than 100 genes known to cause retinal degenerative diseases.
- Discovery of CFH gene — implicated in 50 percent of age-related macular degeneration cases.
- Researchers use gene therapy to successfully treat Usher 1B (animal model).
- Researchers use gene therapy to successfully treat Stargardt disease (animal model).
- Foundation partners with Oxford Biomedica to develop gene therapy for Stargardt disease.
- Phase I clinical trials of gene therapy for LCA begin.
- Nanoparticles show promise for delivering healthy genes to the retina.
- More than 140 genes linked to retinal degenerative diseases.
- Young adults who were nearly blind from LCA gain some vision in Phase I gene therapy clinical trials.
- Gene therapy successful in treating achromatopsia (animal model).
- Foundation establishes partnership with Genable to develop gene therapy for dominant retinitis pigmentosa.
- Foundation partners with AGTC to develop gene therapies for retinoschisis and achromatopsia.
- More than 20 people who were nearly blind experience some vision restoration in three Phase I clinical trials of gene therapy for LCA.