The path to developing gene therapy treatments for retinal degenerations, and other diseases, has been an arduous one. Foundation Fighting Blindness researchers took on much of the early pioneering work in this field and, thanks to their foresight and dedication, gene therapy is emerging as one the greatest breakthroughs in medical research history. These milestones mark the progression of gene therapy for retinal degenerative diseases over two decades.
1995
- First successful gene therapy (animal model).
1997
- Discovery of Stargardt disease gene (ABCR).
1998
- Discovery of Usher 2A gene.
2000
- Gene therapy restores vision in dogs with Leber congenital amaurosis.
2001
- More than 100 genes known to cause retinal degenerative diseases.
2005
- Discovery of CFH gene — implicated in 50 percent of age-related macular degeneration cases.
2006
- 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.
2007
- Phase I clinical trials of gene therapy for LCA begin.
- Nanoparticles show promise for delivering healthy genes to the retina.
2008
- 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.
2009
- 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.
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