Patients With Rare Eye Disorder Getting Experimental Gene Editing Treatment – IOTW Report

Patients With Rare Eye Disorder Getting Experimental Gene Editing Treatment

NPR

Doctors decided to try using CRISPR to edit a gene inside their bodies because retinal cells are too fragile to remove, edit in a lab and try to return to the body. Traditional gene therapy is also impossible for this condition because a healthy version of the defective gene is too big to fit into the genetically modified viruses used to ferry new genes into peoples’ bodies.

Instead, doctors made three small incisions in Kalberer’s right eye and in Knight’s left eye so they could infuse billions of copies of a harmless virus. Each virus had been engineered to carry genetic instructions to manufacture the CRISPR gene-editor inside their retinas.

“It was a bit scary,” says Knight. “It was exciting and scary at the same time.”

The hope was the CRISPR would act like a microscopic surgeon, literally slicing out the genetic mutation in cells in their retinas. That should trigger production of a protein that could restore the function of the light-sensing cells in their retinas, preventing any further loss of vision and possibly restoring at least some of their lost vision. More

4 Comments on Patients With Rare Eye Disorder Getting Experimental Gene Editing Treatment

  1. This is what I’ve been worried about by getting the Kung Flu vaccine.

    Told my wife to put a bullet in my head if CNN started making sense, or if I ever want to watch “The View”.

    She wants that in writing. 🤔

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