The promise of curing genetic diseases by replacing damaged genes with healthy ones is slowly becoming a reality. One recent story is the development of therapy in humans to reverse a form of childhood blindness called Leber congenital amaurosis, or LCA. OregonLive reports on the story of Alexe Webb who, soon after birth, was diagnosed … Continue reading Advancing gene therapy, debunking AR propaganda.
Tag: RPE65
Gene therapy for blindness – when dogged determination pays off!
Leber's congenital amaurosis is a progressive disorder that affects about 3,000 Americans, and hundreds of thousands worldwide, and causes a progressive loss of vision that usually results in blindness. The disease, for which there has until now been no effective treatment, is caused by a mutation in the encoding RPE65, an enzyme which is crucial … Continue reading Gene therapy for blindness – when dogged determination pays off!
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