Crispr Gene-Editing Breakthrough Could Pave Way for Potential Therapies for Genetic Diseases

January 7, 2021

Good news to start a New Year is always welcome, so here we go. 

A team of scientists led by Drs. David Liu and Leslie Gordon used a version of Crispr gene editing to extend the lives of mice with progeria, a rare rapid-aging disease.

Why is this important?
As described in a paper published on Wednesday in Nature and reporting in the Wall Street Journal, progeria is a rare disease that affects only a few hundred children worldwide, but this new research is applicable to developing effective treatments for literally thousands of other genetic diseases that have no effective therapies.

An experimental technique – base editing
Base editing involves “erasing” a single pair of nucleotides in the DNA.

High school biology refresher.  DNA is shaped like a "double helix," which you may remember looks like a twisted ladder.  DNA is made up of pairs of nucleotides designated by the letters A, T, C and G.  A always pairs with T, and C pairs with G.

The tool used in the progeria research is called an “A base editor” because it changes an A-T pair to a G-C pair.  Importantly, unlike other gene-editing techniques, base editing changes DNA without breaking the double helix.  That’s a no-no which can lead to undesirable outcomes.

It’s not just for the kids; there’s business to be had
The research team has been meeting regularly with Beam Therapeutics of Cambridge, Mass., a company co-founded by Dr. Liu that develops base editors for genetic diseases.

Dr. Gordon said progeria paves the way to show potentially that base editing can work in humans which could open a commercial path for other genetic diseases.

  • “There is intense passion by everyone to save the children’s lives,” Dr. Gordon said. “But this isn’t just about a good deed. There is a business case here too.”

A cause for celebration
Correcting the genetic error in the mice is a huge scientific achievement, says Fyodor Urnov, a gene editing expert at the University of California-Berkeley, who was not involved in the new research but is enthusiastic about its potential.

"It's such a cause for celebration.  There's a lot of climbing that lies ahead and a lot of danger and a lot of unpredictability, but the part they just traversed, first, is a breathtaking achievement and second, is the riskiest," he said.

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