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Study: Gene Therapy Can Break Down SCI Scar Tissue
Researchers in London and Amsterdam say a new gene therapy — one that can be activated and de-activated as needed — successfully restored
“hand” function in rats with spinal
cord injuries.
Researchers at King’s College
London and the Netherlands Institute for Neuroscience targeted the scar tissue that forms after a traumatic spinal injury. That scar tissue “prevents new connections being made between nerve cells,” researchers said in a June 14 news announcement.
The gene therapy being studied “causes cells to produce an
enzyme called chondroitinase, which can break down the scar tissue and allow networks of nerve cells to
regenerate.”
Professor Elizabeth Bradbury, Institute of
Psychiatry, Psychology & Neuroscience (IoPPN), said of the new study, “What
is exciting about our approach is that we can precisely control how long the therapy is delivered by using a gene ‘switch.’ This means we can hone in on the optimal amount of time needed for recovery.
“Gene therapy provides a way of treating large areas of the spinal cord with only one injection, and with the switch, we can now turn the gene off when it is no longer needed.”
Dr. Emily Burnside, another one of IoPPN’s researchers, noted that rats and human beings call upon and perform similar series of movements when they are reaching for and grabbing objects — a fact that makes rats valuable to these kinds of studies. “We found that when the gene therapy was switched on
for two months, the rats were able to accurately reach and grasp sugar pellets,” Burnside said. “We also found a dramatic increase in activity in the spinal cord of the rats, suggesting that new connections had been made in the networks of nerve cells.”
Researchers discovered that the rats’ immune systems were able to locate and remove the “gene switch” that was being used to start and stop the gene therapy. Therefore, one of the researchers’ addi-
tional tasks was to figure out a way to overcome that obstacle.
Researchers ultimately solved the problem by adding a ‘stealth gene,’ which effectively hides the gene switch from the immune system so the immune system cannot remove the gene switch.
Because researchers also discovered that a small amount of the gene therapy remained active even after the gene was “switched off,” they’re now working on creating a way to completely stop the gene therapy when it’s no longer needed. Once that issue is addressed, researchers plan to go ahead with trials using larger species of subjects.
The study has been published in the journal Brain. m MobilityMgmt.com
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12 AUGUST2018|MOBILITYMANAGEMENT
SPINAL CORD: ISTOCKPHOTO.COM/JAKARIN2521

