Basic science and animal research drive efforts to preserve patients’ vision
Animals can play crucial roles in helping researchers understand disease and develop new treatments. Without mice, Harvard Medical School geneticist Connie Cepko wouldn’t be on the cusp of discovering whether her team has developed the first safe and effective gene therapy for people with a form of genetic blindness called retinitis pigmentosa.
An estimated 1.5 million people worldwide live with retinitis pigmentosa, which starts with loss of night vision and usually progresses to loss of color, peripheral, and/or central vision. There is currently no cure and no way to stop the disease from advancing.
In this video, Cepko shares her team's approach to developing a gene therapy that aims to preserve vision in people with RP and certain other forms of genetic blindness, including some cases of macular degeneration, by counteracting damage to the retina caused by any of hundreds of different genetic mutations.