Recent advances in ophthalmic genetics and gene therapy were discussed on February 3-4, 2010. Gene therapy clinical trials are currently underway for several medical conditions, including age-related macular degeneration, pulmonary emphysema, prostate cancer, painful diabetic nephropathy, hemophilia B, thyroid tumors, oral and maxillofacial malignant tumors, head and neck malignant tumors, Alzheimer's disease, non-small lung cancer, Tay-Sachs, rheumatoid arthritis, cystic fibrosis, HIV, Huntington's disease, muscular dystrophy, Parkinson's disease, severe combined immunodeficiency syndrome.