incredible! Gene therapy repairs spinal cord injury

Release date: 2018-06-20

The spinal cord is like a network cable in the human body. Once damaged, the affected limbs will be “unstable in the network” or even “broken off the net”, causing discomfort in exercise capacity and even consequences. Every year, countless spinal cord injuries are caused by car accidents or other causes, and the lives of these patients are on the road.

This week, a study published in the neurological journal Brain, let us see the hope of repairing spinal cord damage. Scientists from King's College London have shown that they use gene therapy to restore a spinally injured mouse to a once-damaged athletic ability!

â–²This research has been reported by well-known media such as BBC (Source: BBC official website screenshot)

Let me talk about the spinal cord injury. After the injury, the glial cells secrete a special polysaccharide. It's like crusting after our skin hurts. The problem, however, is that such polysaccharides affect the plasticity of the neurons and thus the connections between the neurons. In this way, the "network cable" can not be connected even if it wants to connect.

To this end, the researchers designed a novel immunotherapy. They plan to send the gene encoding "chondroitin" into the damaged spinal cord, degrading the secreted polysaccharide and restoring neuronal plasticity. This program sounds simple and straightforward, but there are several technical bottlenecks in practice. First, we have no control over the expression levels of these genes. If the expression is too low, it will not work; if the expression is too high, other problems may occur. In addition, after a spinal cord injury, the body's sensitive immune system will mercilessly inhibit foreign gene therapy and affect their effects.

â–² Traditional (left) and new (right) gene therapy design (Source: "Brain")

To overcome these two bottlenecks, the research team has cleverly designed gene therapy—they put a switch on gene therapy that can only be activated when induced. This means we can control it to produce the right level of chondroitin at the right time. In addition, they also camouflaged this gene therapy, allowing it to escape the immune system attack.

“Our approach allows for precise control of the duration of gene therapy through switches. This shows that we can optimize recovery time,” said Professor Elizabeth Bradbury, head of the study. “Gene therapy can be treated with just one injection. Spinal cord damage. The switch that controls gene therapy allows us to turn it off when it doesn't require gene therapy."

In the mouse experiment, this treatment received good results. In the study, normal mice actively extended their right hand, accurately grasping the jelly beans, and stuffing the food into the mouth.

â–² Ordinary mice can accurately grasp the sugar beans (Source: "Brain" video screenshot)

However, mice with spinal cord injuries will have a reduced ability to exercise. Under the temptation of food, they still reach out to grab food. However, the damaged spinal cord made their movements tremble, and they could not accurately catch the jelly beans and even the jelly beans.

â–² spinal cord injured mice catch unstable sugar beans (Source: "Brain" video screenshot)

Under the influence of gene therapy, the researchers found that the spinal cord damage of the mice was improved. In just a few weeks, the mice's simple motor skills have recovered. After about 8 weeks of treatment, the mice's complex athletic ability has also improved significantly - they have regained the ability to accurately capture jelly beans.

â–² After the gene therapy treatment, the mouse recovered the accurate grasping ability (Source: "Brain" video screenshot)

"Our discovery is very exciting. The most important thing for patients with spinal cord damage is to restore their hand function," Professor Bradbury added. "Catch a coffee cup or hold a small toothbrush." It has a huge impact on the quality of life of patients."

At present, this therapy has not yet entered the human clinical trial stage. Researchers are looking forward to confirming its safety and ensuring that it does not cause side effects. Next, they will repeat this experiment in large animals. We look forward to making good progress in these experiments and benefiting the world's spinal cord patients as soon as possible!

Source: Academic Jingwei

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