Gene Therapy For Blindness
A new gene therapy may soon be approved to treat a rare genetic form of vision loss and blindness.
Gene therapy for blindness. The trial a first in human case for x linked retinitis pigmentosa rp was led by dr. Experimental gene therapy for blindness considered by fda. Gene therapy for blindness.
Results from those studies could help determine which conditions are candidates for gene therapy treatment. The research suggests the experimental gene therapy is. But how does it work.
12 a panel of advisers for the food and drug. Robert maclaren at the university of oxford but spanned multiple centers including the bascom palmer eye institute in miami which previously championed luxterna the first fda approved gene therapy for a type of inherited blindness. In december 2017 the fda approved luxturna the first ever gene therapy to treat a genetic disease causing blindness.
1 inserm umr s 968 upmc university of paris 06 institut de la vision paris france. The daily mail also covered the story and said that an injection of genes directly into the retina could reverse blindness in hundreds of thousands of patients. In october he told an fda advisory committee that the first 12 years of his life were spent in darkness.
Wider potential for gene therapy. A year later the therapy was approved in europe with the help of novartis. A long list of retinal dystrophies including retinitis pigmentosa stargardt disease leber congenital amaurosis achromatopsia x linked retinoschisis usher syndrome and choroideremia can be traced to inherited changes in dna.
With the first gene therapy for blindness in the market the door has been opened for a new wave of gene therapy treatments with the potential to cure genetic blindness.
