'Genetic scissors' used to research potential treatment for rare progressive disease

"Genetic scissors" provided hope to a man living with a progressive disease.


Like his father, Bill Rose lives with ATTR amyloidosis.

Rose, who was diagnosed with the genetic protein disorder more than a decade ago, now uses a wheelchair.

"I'm pretty numb from the elbows down and from the knees down and the muscles are wasted in my hands so they don't work properly," he said. "To me, the thing I miss most in my whole life is being able to just stand up and bear hug my wife."

His father didn’t have many treatment options, and his health quickly deteriorated.

But a clinical trial gave Rose hope that things could be different for one of his sons, who also has the gene for the disorder. It involved technology called CRISPR/Cas9, or CRISPR for short.

"So when you think of certain genetic disorders, if we can isolate the gene that causes it," said John Leonard, president and CEO of Intellia Therapeutics. "CRISPR allows us to start thinking about ways to repair that gene and potentially reverse and cure the disease."

Leonard said the class of enzymes could fix broken genes by acting like genetic scissors.

He compared the nucleotides of the human cell DNA to beads in a necklace.

"If you want to find 20 beads in a row out of those 3 billion, you're able to do that with CRISPR/Cas9," Leonard said. "And what that does is that takes the scissors there, which is the enzyme, and alters that DNA in ways that we want it to be altered."

By the end of 2020, the company had started the first in-human clinical trial, something both Leonard and Rose were hopeful about.

"That is the power of CRISPR," Leonard said. "That we can imagine things that we couldn't imagine before."

Rose added, "I just want people to know that the challenge is to focus on the positive. And also to realize that science is progressing and we're really making progress with this."

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