Gene therapy, hope of vision for LCA patients

Gene therapy, hope of vision for LCA patientsRecent study has offered hope to patients living in darkness due to Leber’s congenital amaurosis (LCA). The study has shown that vision of these people can be restored with the help of gene therapy. Leber’s congenital amaurosis (LCA) is a condition, which sets on in childhood and it can lead to total blindness at age 30 to 40.

Researchers at the University of Pennsylvania’s School of Medicine treated 12 patients aged between eight and 44 with the experimental gene therapy. Out of these, five were children and seven were adults.  

During the therapy, the genetic material was injected into the worst eye of each participant. The genetic material was carried piggyback on a virus to the defective eye cells.

All the patients responded well to the treatment. The study subjects were able to walk through a low-light obstacle course. Vision of half of the study subjects was restored enough that they can’t be classified as legally blind. The maximum improvement was shown by the children aged 8, 9, 10 and 11.

Co-author of the study and director of the Centre for Cellular and Molecular Therapeutics, Katherine High, said: “This result is an exciting one for the entire field of gene therapy. This study reports dramatic results in restoring vision to patients who previously had no options for treatment. These findings may expedite development of gene therapy for more common retinal diseases, such as age-related macular degeneration.”