Neurodegenerative diseases, like amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), do not currently have an effective treatment – much less a cure. At this point in time, they’re both fatal and irreversible, leaving patients and their families devastated by the diagnoses.
Dr. Claire Clelland of the University of California San Francisco hopes to change all that, however, by finding a cure for dementia. While working hard in her lab to hit on key discoveries, she’s turned toward the curative potential of CRISPR gene editing. Through this technology, she aims to edit the affected genomes to cure genetic variants of FTD – and ALS as an extension.
Her recent research paper outlines the findings, but it’s just the first step in a long process of proving the treatment works safely. Human trials could still be many years down the road, although patients are hopeful for an accelerated timeline.
What is CRISPR Gene Editing?
CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, allows people to rewrite sequences in the genome. In theory, this process can cure diseases caused by single gene mutations, like both ALS and FTD.
The CRISPR technology first came about through the collaborative work of Professor Jennifer Doudna and Professor Emmanuelle Charpentier. Their discovery earned them the 2020 Nobel Prize in Chemistry, while opening the doors to new developments all across the healthcare world.
Unfortunately, CRISPR gene editing does have its opponents, many of which don’t believe it’s ethical to alter human genes. Their arguments mostly center around changing the genes in infants and children, especially after scientist He Jiankui edited the genomes of three embryos to make them resistant to HIV. Although the embryos produced three healthy babies, demands for more oversight rang out across the scientific community.
In the past few years, researchers in labs all over the world have revealed CRISPR’s potential to treat serious diseases, like diabetes, cancer, and HIV/AIDS. As that occurred, the global scientific community became much more accepting of the technology and all its possibilities.
How CRISPR Can Help Treat Dementia
As an integral part of the UCSF Memory & Aging Center team, Clelland regularly sees the impact dementia has on people. From their ability to control emotions and behavior to retaining their personality and language, the disease has a profound impact on their everyday lives. Her experiences have inspired her to work on finding a cure for patients affected by this detrimental disease.
To do her CRISPR gene editing research, she has to work in the affected cell types made from lab-created stem cells. To create these highly flexible stem cells, researchers start with ordinary cells from the skin or blood. Then, they transform them into the pluripotent state common in the embryonic stage, allowing them to remain at the ready for therapeutic use.
While working within those transformed cells, she’s attempting to eliminate the gene mutations causing FTD and ALS using CRISPR technology. Her initial research shows great promise, although human trials are still needed to confirm the findings. Patients are already voicing their hope about getting a chance to sign up for the trials. Unfortunately, human trials take a long time to set up, putting them several years down the line at best.
Potential for ALS Cures with CRISPR Gene Editing
Since both ALS and dementia often occur due to single-gene mutations, dementia treatment advancements give hope to ALS patients, too. So, while Clelland is not yet working on curing ALS, her research could open the doors to that happening.