MIAMI. A groundbreaking gene therapy, delivered through eyedrops, has successfully restored the sight of a 14-year-old boy, Antonio Vento Carvajal, who had been legally blind for most of his life due to a rare genetic condition called dystrophic epidermolysis bullosa. The therapy not only improved Antonio’s vision but also holds promise for treating millions of people with various eye diseases.
Antonio’s condition caused blisters to form all over his body, including his eyes, leading to severe scarring that affected his vision. Dr. Alfonso Sabater, a physician involved in Antonio’s treatment, observed the positive results of a gene therapy clinical trial for skin lesions and wondered if a similar approach could help Antonio’s eyes.
With the help of drugmaker Krystal Biotech, the treatment, called Vyjuvek, was adapted for Antonio’s eyes using an inactivated herpes simplex virus to deliver functional copies of the gene responsible for producing collagen 7, which is vital for the health of skin and corneas. The treatment was administered through eyedrops, and within two years, Antonio’s vision showed significant improvement.
The innovative gene therapy, which received “compassionate use” approval from the U.S. Food and Drug Administration, offers hope for many other eye diseases beyond Antonio’s condition. The eyedrops could be tailored to address different genetic causes of eye disorders. Dr. Aimee Payne, a dermatology professor at the University of Pennsylvania, lauded the approach, as it targets the root cause of the disease and could potentially benefit numerous patients in the future.
Antonio’s restored vision has been life-changing, allowing him to participate in typical teenage activities like playing video games with friends. The successful outcome of this gene therapy has brought joy to Antonio’s family and has opened the door to exploring similar treatments for other eye-related conditions.
While gene therapy is still a relatively new frontier, its potential to revolutionize medical treatments and improve the lives of countless individuals, like Antonio, is generating excitement among researchers and medical professionals alike. The hope is that with continued research and refinement, gene therapy could offer new avenues of treatment for a wide range of genetic and hereditary diseases in the years to come.
Si Venus L Peñaflor ay naging editor-in-chief ng Newsworld, isang lokal na pahayagan ng Laguna. Publisher din siya ng Daystar Gazette at Tutubi News Magazine. Siya ay isa ring pintor at doll face designer ng Ninay Dolls, ang unang Manikang Pilipino. Kasali siya sa DesignCrowd sa rank na #305 sa 640,000 graphic designers sa buong daigdig. Kasama din siya sa unang Local TV Broadcast sa Laguna na Beyond Manila. Aktibong kasapi siya ng San Pablo Jaycees Senate bilang isang JCI Senator.
