|Professors Woo Se-joon and Professor Joo Gwang-sic|
Researchers at Seoul National University Bundang Hospital have published research on the hereditary retinopathy’s current and future treatment.
The research team, led by Professors Woo Se-joon and Joo Gwang-sic of the department of ophthalmology, disclosed the results of the current development of therapeutic methods and treatments for hereditary retinopathy diseases.
The team also presented the status, limitations, and future directions of genetic retinal disease drugs approved by the U.S. Food and Drug Administration (FDA).
Hereditary retinopathy is a disease caused by various genetic anomalies, which often can lead to blindness and visual field impairment. The illness affects one in every 3,000 people.
Treatments include drug therapy, gene therapy, stem cell therapy, and artificial retinal implantation. However, gene therapy is the only treatment that can fundamentally treat the disease.
Spark Therapeutics’ Luxturna is currently the only hereditary retinopathy gene treatment that has received approval from the FDA.
However, the drug has yet to prove its total efficacy on patients and has a limited patient pool. Also, the price of the prescription price is high giving economic burden to patients.
The team suggested a novel fusion gene therapy system that combines the advantages of viral and nonviral gene therapy as a way to overcome the limitations of gene therapy. The team also confirmed that such method could be safer when compared with conventional gene therapy.
Professors Woo and Joo also stressed that gene scissors and stem could help cure dominant hereditary retinal diseases, which is currently incurable.
“Through the approval of gene therapy products, many patients with hereditary retinopathy have the hope of avoiding blindness," Professor Woo said. “We hope that our journal will lead to continued clinical trials and development of more gene therapy products."
The results of the research were published in Progress in Retinal and Eye Research, a well-known ophthalmology journal.
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