Treating Hereditary blindness by gene therapy will be a revolution in medical field
An experimental gene therapy for a rare hereditary form of blindness is a step closer to reality. An FDA advisory panel voted unanimously Thursday to recommend approval of a groundbreaking treatment for people with a faulty gene that impairs vision. It would be the first gene therapy in the U.S. for an inherited disease.
The treatment, which will be marketed as Luxturna, fixes a mutation in the RPE65 gene. It involves a single treatment to each eye, which introduces genetically engineered virus particles carrying a corrected version of the mutated gene. Spark Therapeutics, the treatment’s developer, estimates that 6,000 people around the world could benefit from this treatment. More than 90 percent of the patients treated in the study showed some improvement in eyesight within just a few days of treatment.
Gene Therapy Breakthrough
This is a huge step forward for the field of gene therapeutics. “[O]n multiple fronts, it’s a first and ushers in a new era of gene therapy,” assistant professor of ophthalmology at the Oregon Health and Science University, Paul Yang, told NPR.
Alone, this treatment could also be applied to other formally incurable genetic eye diseases. “There are a lot of retinal diseases like this, and if you added them together it’s a big thing because they are all incurable,” says lead researcher Albert Maguire in an interview with NPR before the hearing.
Other gene therapy clinical trials are currently being held around the world. According to the Washington Post, diseases ranging from hemophilia to Huntington’s Disease, an inherited condition that causes the progressive breakdown of nerve cells in the brain, are being targeted for gene therapy treatments.
As with any medical treatment, successful gene therapy doesn’t come without risk. Still, this is huge progress toward correcting previously incurable conditions. This treatment and others like it have the potential to transform countless lives in the very near future.