Gene therapy for patients with spinal cord injuries

An international team of scientists is continuing research on gene therapy for patients suffering from neuropathy caused by damage to the spinal cord or peripheral nervous system. The results of the new study have been published in the journal Molecular Therapy.

A new study by an international team led by prof. Martin Marsala from the University of California describes a reversal in the perception of neuropathic pain (from a painful state to pain relief) after the administration of a specific gene therapy. "The application of the gene vector is carried out subpially, that is, under the soft membrane of the spinal cord in the area of ​​the posterior horn. In the tested animals, the treatment lasts for at least 2.5 months after the administration of the gene therapy," explains Dr. Hana Studenovská from the Department of Biomaterials and Bioanalogous Systems (IMC CAS).

In a new study, researchers used pigs (Gottingen-Minnesota type in collaboration with the Institute of Animal Physiology and Genetics in Liběchov, and Yucatan type of pig, in collaboration with the University of California, San Diego) and a cynomolgus monkey (in collaboration with the National Primate Research Center in Thailand) as a large animal model. The application of immunosuppressive therapy and the selection of the appropriate kinetics of the released active substance to suppress the immune response in a specific type of large animal model was part of the preparation of each surgery. "In the Department of Biomaterials and Bioanalogous Systems, we prepared polymer microspheres with a suitable active substance, and at the same time, in cooperation with the Institute of Animal Physiology and Genetics in Liběchov, we focused on the kinetics of releasing immunosuppressants and choosing the optimal drug level with regard to the specific type of surgery," adds Dr. Studenovská.

Research institutions participating in this research:

  • University of California
  • Czech Academy of Sciences
  • Slovak Academy of Sciences
  • University of Okinawa in Japan
  • Bogomoletz Institute of Physiology in Kiev
  • National Primate Research Center of Thailand

The Original Article

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