- Researchers are looking at the CRISPR gene-splicing technology to see if it can be used to treat dementia as well as amyotrophic lateral sclerosis (ALS).
- Scientists say the technique could be used to alter DNA and repair gene mutations that cause these diseases.
- The research is in the early stages with human trials not expected for another year or two.
At her lab on the campus of the University of California San Francisco, Dr. Claire Clelland is working to find cures for neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease.
Both are fatal, irreversible diseases that currently have no effective treatment.
However, Clelland has the help of a relatively new weapon: CRISPR, the gene-editing technology.
“We are currently developing CRISPR gene therapies for genetic forms of FTD and ALS. But we have to know what edit will work and deliver that technology,” explained Clelland, an assistant professor of neurology at the university.
“Single-gene mutations cause disease and ALS, and should be curable by editing the genome,” she told Healthline.
Their preliminary findings were outlined in a research paper published last month.
CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, has rocked the science community in recent years.
It was first introduced by Jennifer Doudna, Ph.D., a professor in the departments of chemistry and molecular and cell biology at the University of California Berkeley, and her collaborator, Emmanuelle Charpentier, PhD, a professor at the Max Planck Unit for the Science of Pathogens in Berlin, Germany.
The two scientists were awarded the 2020 Nobel Prize in Chemistry for their research.
With CRISPR, humans now have the power to rewrite the sequences of small regions of the genome and potentially erase certain diseases.
But CRISPR’s introduction was accompanied by both enthusiasm and scorn. While the technology is unprecedented, there are those who believe human genes should not be altered.
Some say that what CRISPR does is “playing God”.
The
In 2018, Chinese scientist He Jiankui used the CRISPR-Cas9 technology to modify the genomes of embryos. His intent was to make them resistant to HIV. The three babies were subsequently born healthy.
But when they learned of what Jiankui had done, China’s State Council
CRISPR is more accepted in the global scientific community now.
It is being used in labs worldwide to study its abilities to treat and possibly cure cancer, diabetes, HIV/AIDS, and blood disorders.
Because of CRISPR, scientists can now enter DNA and make a change that corrects mutations that cause disease.