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article imageAdvances in gene therapy to help paralysis

By Tim Sandle     Jan 7, 2021 in Science
A new study has successfully used gene therapy to cure paralyzed rats. Some degree of limb function was restored to the rats. The success raises hopes that the treatment could be deployed to assist people who are unable to move their limbs.
At the end of the study, it was found that the rats had regained their ability to use their paws and were able to pick up sugar cubes to feed themselves, according to The Independent. The gene therapy trial was conducted at King’s College London, U.K. The focus of the work was to repair damage to the spinal cords of the rodents. The spinal cords of the rats had been purposefully damaged to mimic the damaged sometimes suffered to humans after car crashes.
Quoted by Sky News, Professor Elizabeth Bradbury, one of the principal researchers, stated: "In some of the tests we looked at – such as gripping the rungs of a ladder – the treatment worked within one to two weeks."
Gene therapy
Gene therapy is an important aspects of medicine. The process is designed to introduce genetic material into cells. This is to compensate for abnormal genes or, alternatively, to produce a beneficial protein. In cases where a mutated gene causes a necessary protein to be faulty or to become missing, then gene therapy could work to introduce a normal copy of the gene and hence to restore the function of the protein.
There are different variants of gene therapy, including plasmid DNA, where circular DNA molecules are genetically engineered so they carry therapeutic genes into human cells; viral vectors, where viruses are used to deliver genetic material into cells; bacterial vectors, where bacteria are modified and then deployed as vehicles to carry therapeutic genes into human tissues; and human gene editing technology, where genes are edited to disrupt harmful genes or to repair mutated genes. There is also patient-derived cellular gene therapy products. With this more recent process, cells are taken from the patient, modified and then returned to the patient.
For some scientists, the next phase is germinal gene therapy. This has been achieved experimentally in animals but not in humans.
Novel research
With the new study, the process involved injecting a gene that produces an enzyme called chondroitinase, into the spinal cords of the rats. This enzyme functions to breaks down scar tissue, a tissue that is formed following damage to the spinal cord. he tissue prevents new connections from being formed between nerves. The enzyme is also being used in trials for vitreous attachment and for treating cancer.
More about Spinal cord, Nerves, Gene therapy
 
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