Once upon a time, the most promising medications were mass-produced for a global population. Although these drugs are still critical, modern technology is introducing more personalized options with the help of gene editing. CRISPR gene-editing technology is changing how researchers look at medical care, from cancer treatments to ending the cycle of genetic diseases like sickle cell.
What is CRISPR Gene Editing?
CRISPR gene editing is a revolutionary technology that allows scientists to make precise cuts in the DNA to remove, disable or replace genes. At its core, CRISPR mimics a defense system standard in bacteria.
CRISPR stands for clustered regularly interspaced short palindromic repeats. It is a fancy way of saying the technology can hone in on patterns in genetic code.
For bacteria, these patterns represent pieces of viral code. They can use this code to identify lethal viruses if they invade, allowing them to take a defensive posture. The bacteria send Cas9, a type of enzymatic scissors, to cut pieces of code from attacking viruses. Then, they save that code to use later to recognize these organisms if they return, almost like a mug shot.
CRISPR technology can apply that same concept to identifying segments of DNA. It is an innovative approach to helping those with genetic disorders.
CRISPR and Sickle Cell
Each year, hundreds of thousands of parents pass on at least one medical problem to their child through their DNA. Genetic disorders are mutations in the genes that cause abnormalities. The most common inherited disease in the world is sickle cell.
In the U.S., around 100,000 people have sickle cell disease, and 400,000 babies are born with it each year worldwide. Unfortunately, there is no cure for genetic disorders like sickle cell because the condition is literally encoded in each person’s DNA. That also makes it hard to stop the disease cycle in families.
Experimental treatments using CRISPR are already changing the lives of people with sickle cell disease. Sickle cell affects the hemoglobin in red blood cells. People with it have episodes of intense pain caused by either clots or a shortage of red blood cells. A recent clinical trial was able to flip the switch on sickle cell for 31 sufferers using CRISPR gene editing.
Researchers took blood from each patient and fixed the problem in the DNA. They then transfused the corrected blood back into the patient. As a result, 31 people no longer suffer from the prolonged episodes of pain that are benchmarkers for the disease.
Other Gene Editing Breakthroughs
The game-changing treatment of sickle cell is just one of many stories showing how CRISPR revolutionizes medicine. For example, researchers with biotech firm Intellia Therapeutics recently offered promising data on the effect of gene editing in the treatment of rare genetic liver disease. With the help of CRISPR, scientists could “turn off” the production of a toxic protein.
Researchers are studying the application of CRISPR in many conditions, including muscular dystrophy, HIV, and Huntington’s. Studies are even looking to fix the genetic components that lead to heart disease and high cholesterol. And they may be just scratching the surface of promising applications for CRISPR gene editing.
