Posts about leber congenital amaurosis


Gene Therapy by L.E. Carmichael - Front Cover

Gene Therapy: Vectors, Viruses, and Why CRISPR Will Change Everything

Gene therapy involves repairing or replacing a faulty gene that has led to disease. In order to do that, one major hurdle must be overcome: getting new DNA into the patient's cells. Cells, however, are designed to keep things ... Read More  

Gene Therapy by L.E. Carmichael - Front Cover

Introduction to Gene Therapy: It Sounds Simple, But It’s Sure Not Easy

Muscular dystrophy, hemophilia, Leber congenital amaurosis (LCA), cancer... they all have one thing in common. They are genetic diseases, ultimately caused by missing or malfunctioning genes in the patient's DNA. Until recently, we could treat the symptoms of many ... Read More