Intellia Presents:
CRISPR/Cas9
A Nobel Prize-winning gene editing technology
CRISPR/Cas9 is a Nobel Prize-winning gene editing technology that leverages the body’s natural processes to precisely edit DNA.
OVERVIEW
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.
HOW DOES CRISPR WORK?
The CRISPR/Cas9 system works by targeting a specific location in the DNA to make a change.
HOW DOES CRISPR WORK?
Sections of our DNA make up what we know as genes, which contain instructions for making proteins.
HOW DOES CRISPR WORK?
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.
HOW DOES CRISPR WORK?
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:
CAS9 Enzyme
Cas9 is the enzyme responsible for editing the gene.
Guide RNA
Acts as a molecular GPS for the Cas9 enzyme. Guide RNA locates the precise spot in the DNA that needs editing.
HOW DOES CRISPR WORK?
Once an edit is made, the natural DNA repair process takes place. This is intended to stop production of the disease-causing protein.
Intellia Presents:
CRISPR/Cas9
A Nobel Prize-winning gene editing technology
OVERVIEW
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.
HOW DOES CRISPR WORK?
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:
CAS9 Enzyme
Cas9 is the enzyme responsible for editing the gene.
Guide RNA
Acts as a molecular GPS for the Cas9 enzyme. Guide RNA locates the precise spot in the DNA that needs editing.
Once an edit is made, the natural DNA repair process takes place. This is intended to stop production of the disease-causing protein.
Genome editing research is rapidly leading to the discovery of new investigational technologies
All with one goal in mind: To create a new class of therapies that may address a growing number of diseases where there is unmet medical need.
Our CRISPR Videos