Gene Therapy Milestones

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.