Korea’s First Successful Administration of Retinal Gene Therapy

A team led by Professor Kim, Sang Jin of the Department of Ophthalmology at Samsung Medical Center announced on July 13th that for the first time in Korea, Luxturna, a gene therapy for Leber Congenital Amaurosis, was administered in one eye through vitrectomy. One week later, the surgery was successfully completed on the other eye.


Leber Congenital Amaurosis refers to a disease in which the function of photoreceptor cells of the retina decreases and disappears due to genetic mutations in the retina, resulting in severe vision loss, night blindness and nystagmus from a young age.


Luxturna copies the normal RPE65 gene to the adeno-associated virus, which is made harmless to the human body, and then is administered to the patient's retina to make the normal gene function instead of the mutated gene.


The results of clinical trials in the United States have shown that although it is not possible to restore normal vision after treatment, it is effective in restoring visual function, such as preventing permanent vision loss and enhancing light sensing ability so that you can live independently.


Professor Kim, Sang Jin stated that in Korea, even ophthalmologists conclude that inherited retinal degeneration is an incurable disease and have a passive approach when it comes to attempting genetic diagnosis. He also mentioned that although there is only one gene therapy currently available, many gene therapies are expected to be developed within the next few years.