Gene Therapy Repairs Spinal Cord Damage In Formerly Paralyzed Rats

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A research team from King’s College in London has taken a giant step toward helping paralyzed individuals regain “control of their hands.” In a recent experiment, the scientists have had success repairing spinal cord damage in rats using gene therapy.

The spinal cord is a cylindrical tube of nerve fibers and connected tissue that is enclosed within the spine. It carries instructions to virtually every part of the body, forming the central nervous system.

When the spinal cord has been damaged, the body repairs it with scar tissue. Unfortunately, the scar becomes a barrier, blocking the formation of new nerve connections.

In order to dissolve scar tissue in the rats’ spinal cords, the research team rewrote the spinal cord’s genetic instructions. They utilized a virus to deliver genetic material to code the enzyme chondroitinase. The scientists then gave a drug to activate the expression of the gene within the rats’ bodies.

A previous study completed by Professor Elizabeth Bradbury, published in Nature, suggested that Chondroitinase ABC promotes recovery after injury to the spinal cord. The results of the study demonstrated that “CSPGs are important inhibitory molecules in vivo and suggest that their manipulation will be useful for the treatment of human spinal injuries.”

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According to a report by BBC News, the most remarkable outcome of the study occurred when the research team witnessed the rats regaining the use of their front paws. Researcher Dr. Emily Burnside stated, when the gene therapy was active for two months, the animals were “able to accurately reach and grasp sugar pellets.”

The scientists behind this study also found a “dramatic increase in activity in the spinal cord of the rats,” which suggested new connections in the nervous system were being made.

Professor Elizabeth Bradbury explained that regaining function of this type is an extremely exciting breakthrough. This is because, for individuals suffering from paralysis, regaining hand function is the highest priority.

When a quadriplegic is able to regain control of his or her arms and hands, they are able to mobilize themselves in a wheelchair. Quality of life is greatly increased when an individual who has suffered a spinal cord injury is able to eat, drink, or even brush their own teeth.

The research team hopes that their approach will be a viable treatment for individuals who have been injured in falls or car accidents. Dr. Mark Bacon, a spinal researcher, stated that the data is compelling, however, the approach is not yet ready for human trials.

“It’s exciting, but getting approval for gene therapies represents a particular, but not insurmountable, challenge to getting it to the clinic.”

The European scientists believe that their gene therapy approach could truly be a life-changing option for the millions of individuals who suffer from spinal cord injuries.