CRISPR/Cas9 is a Nobel Prize-winning gene editing technology that leverages the body’s natural processes to precisely edit DNA.
It has been around for billions of years, initially used by bacteria as part of their immune system to ward off viruses. Today, scientists at Intellia are developing this technology to make targeted changes to genes directly implicated in human disease.
In the future, Intellia hopes to use its CRISPR technology to address a variety of severe, life-threatening diseases.
The CRISPR/Cas9 system works by targeting a specific location in the DNA to make a change.
Sections of our DNA make up what we know as genes, which contain instructions for making proteins.
Sometimes, those instructions contain errors and proteins are incorrectly produced, which may lead to disease. Other times, a correctly produced protein can act differently than it is supposed to, also leading to disease.
CRISPR-based therapies have the potential to address diseases at the genetic level. To do this, the technology is made up of two main components:
Once an edit is made, the natural DNA repair process takes place. This is intended to stop production of the disease-causing protein.
In other instances, where a disease is caused by the absence of a required protein, CRISPR-based gene editing is also being explored as a potential therapeutic solution.
To learn more, watch this animation.
The CRISPR/Cas9 system works by targeting a specific location in the DNA to make a change.
Sections of our DNA make up what we know as genes, which contain instructions for making proteins.
Sometimes, those instructions contain errors and proteins are incorrectly produced, which may lead to disease. Other times, a correctly produced protein can act differently than it is supposed to, also leading to disease.
CRISPR-based therapies have the potential to address diseases at the genetic level. To do this, the technology is made up of two main components:
Once an edit is made, the natural DNA repair process takes place. This is intended to stop production of the disease-causing protein.
In other instances, where a disease is caused by the absence of a required protein, CRISPR-based gene editing is also being explored as a potential therapeutic solution.
To learn more, watch this animation.
