Skip to main content

Scientists want human trials for gene therapy that could help battle addiction

In recent years, new gene editing tools have been used for everything from genetic modification of plants to increase crop yields to, far more controversially, genetic tampering with human embryos. Could a form of gene therapy also be useful in helping treat cocaine addiction, a form of addiction that proves highly resistant to alternative approaches, such as conventional medical treatment and psychotherapy? That’s what researchers from the world-famous Mayo Clinic are hoping to prove.

They are seeking approval for the first-in-human studies of an innovative new single-dose gene therapy. Their approach involves the delivery of a gene coding for an enzyme, called AAV8-hCocH, which metabolizes cocaine in the body into harmless byproducts. In order to progress to this next step in their work, they first have to gain permission from the U.S. Food and Drug Administration (FDA) in the form of an Investigational New Drug Application.

The researchers have already demonstrated the safety of their approach in mice. In a prior experiment, they showed a complete lack of adverse effects in mice which had both been previously exposed to cocaine and those which had not.

“Mice given one injection of AAV8-hCocH and regular daily injections of cocaine had far less tissue pathology than cocaine-injected mice with no vector treatment,” the researchers wrote in the abstract for their paper describing the work. “Biodistribution analysis showed the vector located almost exclusively in the liver. These results indicate that a liver-directed AAV8-hCocH gene transfer at reasonable dosage is safe, well-tolerated, and effective. Thus, gene transfer therapy emerges as a radically new approach to treat compulsive cocaine abuse.”

This is not the first time similar work has been carried out. In February 2017, scientists at the University of British Columbia genetically engineered a mouse so as to be incapable of becoming addicted to cocaine. However, one of the researchers on the project told Digital Trends that transferring this work across to humans for possible treatment for addiction was not straightforward. Instead, that work was more focused on exploring the link between drug use and genetics and biochemistry.

There’s still a whole lot more research that needs to be done in this area. Even if the FDA grants the Mayo Clinic researchers permission for their human trials, we’ll most likely be waiting a few years at least before this treatment could be rolled out to the general public. It’s an exciting leap forward, nonetheless.

Luke Dormehl
I'm a UK-based tech writer covering Cool Tech at Digital Trends. I've also written for Fast Company, Wired, the Guardian…
CRISPR gene editing could help stop a common poultry virus in its tracks
crispr gene editing chicken virus

Researchers at the Czech Academy of Sciences may have successfully used CRISPR gene editing to create chickens that are resistant to avian leukosis virus (ALV), a common but deadly affliction that affects poultry with symptoms ranging from emaciation and dehydration to depressed behavior. This latest advance could potentially be a game changer when it comes to chicken welfare, with clear implications for meat and egg production around the world.

The ALV-J subgroup virus binds to a protein called chicken NHE-1 (chNHE-1). Replication of this virus depends on a functional chNHE-1 cellular receptor. In previous work, the researchers showed that it is possible to prevent ALV from infecting chicken cells by deleting three letters from the chNHE-1 gene. But in their newer work, they have created a young rooster with sperm that has the exact chNHE-1 gene deletion. Its offspring was a flock of white leghorn chickens with the deletion in both copies of the gene. “It’s quite simple to do,” Jiri Hejnar, the lead researcher on the project, told New Scientist.

Read more
Scientists want to implant mini human brains in animals
researchers create quad core computer from four rat brains lab

It sounds like something out of a 1980s horror movie, but medical researchers are now able to transplant human "mini-brains," also known as brain organoids, into animals like rats. As gruesome as it may sound to implant human brain tissue into animals, this research provides a way to safely research diseases and health problems we struggle to understand, including genetic disorders that affect the brain.

Recently, a new paper called for the creation of a framework that researchers can use to make ethical decisions about brain organoids. Digital Trends spoke to Dr. Isaac Chen, lead author of the paper and an assistant professor of neurosurgery at Penn Medicine, about the ethical issues involved in this research, as well as its potential benefits.
Growing organoids
The process begins with taking a sample of stem cells from a human. These pluripotent stem cells have the potential to develop into other types of cells, such as neurons, as required within the body, and the scientists use this natural pathway of development to grow the organoid. The cells are grown as a colony, and they naturally ball up into a group to create a small organoid, about the size of a pea.

Read more
CRISPR-Cas9 gene editing could one day ‘turn off’ HIV virus in the body
hiv vaccine therapy daily drugs color

HIV treatment has come a long way over the years, due in large part to antiretroviral drugs that stop the HIV virus from replicating in the body. This gives the immune system a chance to repair itself and stop further damage. Thanks to these amazing advances, HIV is no longer the death sentence that it was in previous decades.

However, antiretrovirals only keep HIV at bay for as long as they’re taken. Defaulting on the drugs means that the HIV virus comes back. Even worse, it can cause patients to build up resistance to the antiretrovirals so that they do not work so effectively in the future.

Read more