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Posts tagged rods

How Evolution is Leading to Gene Therapies for More Retinal Diseases

Dr. John FlanneryAt first blush, gene therapy for retinal diseases seems so simple: Inject a tiny drop of liquid containing good copies of a gene to replace the bad, and you’re home free. Vision is saved, and, in some cases, it’s even restored.

But the reality is: Developing gene therapies that are safe, effective and long-lasting is very challenging in our world of genetically diverse retinal degenerations. Scientists have to design a delivery system that gets the genes to the right types of cells across the entire retina, but without affecting other cells.
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New Clues for Keeping Cones Alive

Retinal cells, with cones in blue.Woody Allen once said, “I’m not afraid of death; I just don’t want to be there when it happens.” However, for researchers fighting blindness, “being there” when cones die — at least the cones in a mouse model of retinal disease — was recently a good thing. It provided important clues about how to keep them alive in people.

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