Cellular gene therapy of spinal cord injuries
KFU scientists have successfully applied a combination of two genes in the therapy of spinal cord injuries
Natalia Doroshkevich, KFU Press Service
Researchers at Kazan Federal University have embedded in umbilical cord blood cells a combination of two genes responsible for the production of factors that can support not only the survival of neurons, but also stimulate the growth of axons and new vessels. The resulting gene-cell structure was transplanted into the area of spinal cord injury in a rat, which caused a powerful therapeutic effect in the animal – from improving motor activity (the ability to move the hind limbs, control the paws when walking) to restoring damaged nerve fibers.
The results of the scientists' scientific work were published on March 22 in the journal PlosOne (Mukhamedshina et al., Assessment of Glial Scar, Tissue Sparing, Behavioral Recovery and Axonal Regeneration following Acute Transplantation of Genetically Modified Human Umbilical Cord Blood Cells in a Rat Model of Spinal Cord Contusion – VM).
Earlier, when combining cell and gene therapy, scientists inserted one therapeutic gene into the umbilical cord blood cells (an article with the results of the work was published in the journal Spinal Cord on September 29, 2015).
"It is important that no rodent has developed an extensive post–traumatic cavity in the area of injury, which usually appears at the place of application of mechanical force (impact)," Yana Mukhamedshina, researcher at the Virtual OpenLab Gene and Cell Technologies, told about the study. "The area of nerve tissue damage was filled with regenerating nerve fibers (neuron processes) and transplanted stem cells."
As you know, spinal cord injury remains one of the most pressing medical and social problems. There are no effective methods of its treatment, which is the reason for the appearance of an increasing number of people who drop out of public life due to disability.
Currently, the possibility of overcoming the consequences of spinal cord injury is associated with the use of stem cells and genetic engineering. So, scientific groups, both of the world's leading universities and small laboratories, are searching for effective means of restoring lost functions after spinal cord injuries (first of all, movement and sensitivity). Most of them associate the possible success in treating the consequences of spinal cord injuries with stem and progenitor cells, which are a rich source of various trophic factors that have a positive effect on the survival of neurons and other cells of the nervous system.
The efforts of scientists at Kazan Federal University are aimed at finding the most effective and safe gene-cell drug for the treatment of traumatic spinal cord injuries.
"This problem is acute, because most of these patients become disabled and often drop out of society," says Yana Mukhamedshina. – The world experience of the last decades shows that preclinical trials with stem cell therapy, which are carried out most often on small animals: rats and mice, show extremely positive and inspiring results in restoring the structure and functions of various tissues, including the injured spinal cord. However, the transfer of the experience gained to the clinic is very slow due to concerns about the safety and effectiveness of this therapy for humans."
That is why, before conducting clinical trials, the effectiveness and safety of treatment will be tested on large animals – primates and pigs. Scientists emphasize that if these studies show results similar to positive tests on rats, it will be possible to count with more confidence on the restoration of lost functions in humans.
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11.04.2016