SEP 20, 2019 8:21 AM PDT

Can CRISPR Technology Treat Alzheimer's?

WRITTEN BY: Annie Lennon

New drugs that seek to treat Alzheimer’s disease (AD) have a 99.6% failure rate. This makes it the only disease in the top 10 causes of death that cannot be prevented, slowed or cured. To this end, there is a lot of hope in new technology to finally overcome these hurdles. CRISPR technology in particular, with its ability to edit genes, is particularly promising. 

Yet, despite all the hype around CRISPR technology, the Food and Drug Administration (FDA) has only approved one CRISPR-related treatment so far, and not for AD. Instead, it seeks to treat various types of cancer, having entered clinical trials in May with the hope of measuring results from 18 patients (Fan: 2019). But why aren’t there any clinical trials to treat AD?

Creating a CRISPR technique to treat conditions of the central nervous system in people by itself poses many challenges. In particular, neuroscientists have struggled to find a safe and effective way to deliver CRISPR technology to targeted cells due to difficulties in designing a treatment that can safely cross the blood-brain barrier, traverse organelles and eventually end up in the targeted cell nuclei work (Fernandez: 2019). Only adding to this is the limited knowledge researchers have on the brain itself in comparison to other regions of the body, making researchers even more cautious of the technique (Rudin: 2018). 

Despite these difficulties however, CRISPR technology has nevertheless had some breakthroughs in possible treatments for AD. For example, a team of Korean researchers have created nanoparticles that act as delivery drones for transporting CRISPR to brain cells in people with Alzheimer’s. Their treatment works by turning off the Bace1 gene, known to stimulate amyloid beta production, which has been linked to Alzheimer’s cognitive effects (Park: 2019). 

So far, it seems to work well. After a single dose of the treatment, mice with characteristics of AD experienced significant reductions in their beta-amyloid levels, alongside signs of improved memory and learning. Although promising, the researchers feel that before human trials could begin, they need to confirm their results on higher primates too (ibid.). 

Meanwhile, CRISPR technology is also being used to advance our understanding in other areas of AD treatment. Researchers at the Institute of Molecular Pathology Biomarkers (IBPM) for example used CRISPR technology to identify a biomarker for the disease. Finding a deficiency in the protein STIM1 in the brain tissue of those with AD, they used CRISPR to silence the gene that allows STIM1 to be expressed to see whether a lack of the protein could be linked to Alzheimer’s. When inactivated, the researchers noticed the development of AD-like patterns, confirming a link between STIM1 and the disease, thus creating new avenues for research and eventual drug development (University of Extremadura: 2018). 

To conclude, although CRISPR shows promising signs for one day treating AD, further research on both how the disease functions, as well as how CRISPR may be delivered is required before treatments can properly be developed. In the meantime however, it is clear that the technique may still be used to develop our understanding of the disease and explore possibilities with other treatments too. 

 

Sources

 

Fan, Shelly: Singularity Hub 

Fernandez, Tara: Massive Science

Rudin, Mark: Idaho Statesman

University of Extremadura: Eureka Alert!

Park, Hanseul: Nature

About the Author
  • Annie graduated from University College London and began traveling the world. She is currently a writer with keen interests in genetics, psychology and neuroscience; her current focus on the interplay between these fields to understand how to create meaningful interactions and environments.
You May Also Like
NOV 17, 2019
Neuroscience
NOV 17, 2019
Finding Pleasure in Music Comes From Balance of Uncertainty, Surprise
  A new study by the Max Planck Institute for Human Cognitive and Brain Sciences shows the effect that uncertainty, about a song's progression, an...
DEC 01, 2019
Neuroscience
DEC 01, 2019
Are teens addicted to their phones?
Journalists quickly jumped to the conclusion that 23% of teenagers are addicted to their smartphones thanks to findings from a new study from King’s ...
DEC 04, 2019
Neuroscience
DEC 04, 2019
Single Dose Ketamine Could Rewire Alcohol, Drug Dependance
Presentation by Tobias Stephenson about previous research exploring ketamine as an addiction treatment.    Researchers at University College Lond...
DEC 22, 2019
Drug Discovery & Development
DEC 22, 2019
Effective Therapeutic Approved for Migraines
The U.S Food and Drug Administration has now approved a new medication for migraines. The drug is called ‘ubrogepant (Ubrelvy)’ and comes in th...
DEC 30, 2019
Genetics & Genomics
DEC 30, 2019
Chinese Scientists Implant Genes for Human Intelligence in Monkeys
Scientists from China and the US have implanted a human gene linked to intelligence in the genomes of macaque monkeys. The first experiment of its kind, th...
FEB 18, 2020
Neuroscience
FEB 18, 2020
The Wearable that Spots Early Signs of Alzheimer's
Since 2000, the prevalence of Alzheimer’s has increased by almost 90%. With an estimated 5.8 million Americans suffering from the disease, the Early ...
Loading Comments...