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Gene Therapy – Can It Save Children Living with SMA?
The spinal muscular atrophy (SMA) is a rare genetic disease that can severely affect the quality of life of children. The disease primarily involves damage to the motor neurons in the spinal cord, leading to muscle weakness and even muscle atrophy. This process hinders the normal development of movement and, in severe cases, can cause respiratory muscle failure, necessitating mechanical respiratory support. Early diagnosis of SMA is crucial, as timely initiation of treatment can result in significant improvements in the quality of life for young patients. Until now, the disease has been virtually untreatable, with the worst cases not allowing children to survive past their first year. However, the latest…
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Treatment of Spinal Cord Injury Using mRNA Technology
The treatment of spinal cord injuries presents a significant challenge for science and medicine. The inflammatory processes that occur as a result of the injuries can damage not only the injured area but also the surrounding healthy tissues. Therefore, it is crucial to perform effective interventions as soon as possible. The time factor plays a prominent role in the success of recovery, as delays can further worsen the injured condition. To reduce inflammation and promote regeneration, numerous research efforts are underway worldwide. The Neuroregeneration Laboratory at the University of Szeged, led by Professor Antal Nógrádi, has been working for years on more effective treatments for spinal cord injuries. By applying…
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The Potential of Gene Therapy
The treatment of genetic disorders is one of the greatest challenges in medicine. Traditional therapies, which typically focus on alleviating symptoms, do not address the problem by eliminating the underlying causes. However, scientific research is continuously evolving, and more and more promising solutions are emerging, showing progress in the fields of gene therapy and gene editing. Gene therapy aims to restore faulty genes or improve their function. This process is generally carried out through molecular biological techniques that allow new genes to be introduced into the cell nucleus. A gene, located in the cell nucleus, refers to a specific segment of DNA that plays a key role in regulating life…
