New research out of Illinois is showing promise for people who are suffering from Huntington’s Disease and Amyotrophic lateral sclerosis (ALS), thanks to the help of CRISPR gene editing. Keep in mind that research is still in the mouse phase before any tests can be done on humans.
About ALS and Huntington’s Disease
ALS strikes the nerves that control muscles in the body, eventually causing the nerves to cease functioning and eventually fail. Under such circumstances, people grow weak and become paralyzed.
In the United States, people often refer to ALS as “Lou Gehrig’s disease” for the baseball player who was forced to retire from the game after being afflicted by this condition.
According to the Centers for Disease Control and Prevention, approximately 12,000-15,000 individuals have ALS in America. People tend to be diagnosed with ALS between the ages of 55 and 75, typically living as many as 5 years after the diagnosis, per the CDC.
Huntington’s disease is a condition where neurons in a person’s brain degenerate, which leads to such problems as loss of intellect, the inability to control your movements, and emotional outbursts.
More than 15,000 people in the U.S. currently suffer from Huntington’s disease, according to a report from Johns Hopkins. Individuals with Huntington’s disease will perish from this ailment within 20 years after symptoms first appear.
Scientists Test CRISPR Gene Editing for ALS and Huntington’s Disease
At the University of Illinois Urbana-Champaign, scientists have achieved success in an experiment designed to deactivate mutation of the proteins that lead to ALS and Huntington’s disease. They tested their idea on mice’s central nervous systems in a lab.
According to Tech Explorist, the scientists used a form of CRISPR gene editing known as CRISPR-Cas13 in their experiments on mice that are bred to have genetic mutations implicated in causing Huntington’s disease and ALS. They are using the CRISPR-Cas13 approach to focus on messenger RNA.
The Cas13 system seeks out and cuts the RNAs that result in mutant proteins leading to the two nervous system diseases in question. One reason the scientists are using this version of CRISPR is that by focusing on RNA, the effects produced inside a person’s cells can be reversed. This is said to be due to RNA being a transient molecule.
A gene that many scientists are aiming at is called Superoxide Dismutase 1 (SOD1). After the researchers in Illinois injected CRISPR-Cas13 genetic engineering systems into the mice’s central nervous systems, they discovered that the procedure cut down on the level of mutated proteins in their nervous system for ALS as well as Huntington’s disease.
According to the researchers, “The reduction in mutant SOD1 protein also correlated with better therapeutic outcomes: Mice with ALS that received the CRISPR-Cas13 injection had slower disease progression, improved survival and a slower rate of decline in grip strength and motor skills compared with mice that did not receive the treatment.”
What remains to be seen now is whether using Cas13 CRISPR gene editing will work in human beings as well as it appears to be doing with mice in a laboratory.
New Hope for Patients With ALS and Huntington’s Disease Thanks to CRISPR Gene Editing?
From the 1950s when scientists started describing DNA to the early advances in genetic engineering in the later half of the 20th century to today’s use of CRISPR gene editing, there’s never been a more exciting time to be involved in research into these ailments of the nervous system.
Patients and their friends and families will be eagerly looking forward to follow-up studies to test how good of a tool CRISPR may be for editing genes and correcting defects that lead to ALS and Huntington’s Disease.