Does Gene Editing Offer a Pathway to Improved Mental Health?

Like any new scientific development, CRISPR is often in the news for all the wrong reasons. Don’t get me wrong, there are some very good reasons to fear gene editing, but it’s not all designer babies. Gene editing holds real promise as a revolutionary medical treatment. Imagine deleting the genes responsible for the many heritable diseases that cause devastating pain; it’s perhaps the most efficacious preventative measure possible to take, and its application is only growing. 

Developments are constant, and research into how this technology can be applied to treat psychiatric disorders with a genetic basis is incredibly new, so whether you’re working in the US healthcare system, enrolled in any medical programs, online post master’s PMNP program, or simply want to learn more about promising research and mental health. This article is your guide to CRISPR’s cutting-edge developments. 

What is CRISPR?

CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a revolutionary gene editing technology. It was adapted from a natural system in bacteria and allows precise editing of DNA. CRISPR lets us pinpoint exact parts of the DNA sequence and remove or change them. Its main use is to edit DNA, but it can also be used to regulate genes by turning them on or off without altering their sequence. 

The technology relies on ‘Cas’ proteins, especially Cas9, which can be programmed to target almost any DNA sequence. These proteins cut the DNA so it can be modified or replaced.

CRISPR has made gene editing more affordable and accessible, with applications in both plants and animals. It promises major advances in scientific research and medicine, including the potential to treat and prevent many diseases. This technology is so groundbreaking that in 2020, CRISPR-Cas9 was given a Nobel Prize in Chemistry. 

1. Mental Health Applications

Anyone up to date with CRISPR will know of its applications for cancers, rare eye diseases, or sickle cell; you likely haven’t heard of how gene editing can be used to manage and treat mental disorders:

2. Anxiety 

Imagine a purely genetic solution to anxiety–and well, you wouldn’t be far off the mark. A study published in 2023 looked at how CRISPR could be used to modulate the HTR-2A gene. This gene, in essence, encodes for a serotonin receptor that mostly affects the brain. Mutations in the HTR2A had been observed in schizophrenic and obsessive-compulsive disorders and had been associated with a response to some antidepressants, which work to increase serotonin levels.  

The real challenge in developing treatments like this is the blood-brain barrier, which must be crossed to treat this central nervous system disorder. To do this, the researchers deliver the drug intranasally or up the nose–into mice. 

The finding overwhelmingly supported the theory that regulation of the 5HT-2A gene could indeed reduce anxiety; out of two populations of mice, anxiety-like behaviors decreased significantly after the treatment had been given. 

3. Post Traumatic Stress Disorder 

PTSD is a condition that affects over 3.5% of adult Americans every year, and leading research shows that there is a significant genetic component to PTSD, with 95 specific locations in the genome linked with a risk of developing the disorder. Unfortunately, trauma early in life is one of the greatest indicators of psychiatric issues later in life; however, there is clearly a genetic component as well. Studies have been focused on how the expression of certain genes affects our life outcomes, even associating early experiences of threat with faster aging. There is a range of ways in which we can use CRISPR to intervene. 

Although research is ongoing, a recent study found that heavy alcohol consumption during adolescence can lead to epigenetic changes. One specific gene affected is Arc (activity-regulated cytoskeleton-associated protein), which plays a crucial role in brain function. When alcohol exposure occurs during adolescence, it messes with the enhancer region of the Arc gene. Enhancers act like switches, determining when and how much a gene is turned on or off.

Now, here’s where it gets interesting: scientists used specialized tools called dCas9-P300 and dCas9-KRAB to manipulate the epigenetic marks on the Arc gene. Picture these tools as tiny editors fine-tuning the gene switches. With dCas9-P300, they boosted histone acetylation at the Arc enhancer, essentially turning the gene on. This normalized Arc expression. On the flip side, dCas9-KRAB increased repressive histone methylation, turning the gene “off.” The outcome? By adjusting these gene switches in the amygdala, researchers improved mental health outcomes in rats exposed to alcohol during adolescence. Sort of like tuning a radio station for better reception. 

4. Genetic Solutions for ‘Mental’ Problems

Only time will tell if and when we will get a public and affordable solution via CRISPR; as of today, there are only two FDA-approved treatments that use CRISPR–both for sickle cell disease and cost around 2.2 million. All the research shows that gene editing has incredible promise, and the fact that treatments using gene editing have already been approved is incredible, considering that we only mapped the human genome in 2003.