Today, the an FDA advisory committee recommended that the FDA approve full clinical trials for a type of gene therapy that addresses a rare genetic condition causing deterioration of the retina. This is found in 8.6×10⁵ of people world wide, so not many. the therapy involves injecting a virus bearing the preferred copy of the gene, the non-broken allele, into the eyeball, where the new gene somehow reduces, stops, and seemingly reverses, the deterioration.

The therapy was previously looked at in a preliminary study with a small sample of people. Here is the abstract from that study: Continue reading Gene Therapy Is Starting To Be A Real Thing →