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Gene Therapy Promotes Nerve Regeneration

By: Sai Srihaas Potu

Over the last couple of decades, gene therapy has become one of the most studied topics in the field of genetics. With the potential to cure some of the most prevalent diseases known to mankind, gene therapy can help save millions of lives. Back in 1972, scientists Friedmann and Roblin first discovered gene therapy in order to fix genetic mutations. However, as the years have gone by, the role of gene therapy in the medical field has only grown. The remarkable exploration in the field of genetics has allowed researchers to fully understand the potential of gene therapy.

Genetic therapy is an experimental technique that fixes genetic mutations through the insertion of the correct gene form. Through gene therapy, researchers will be able to better understand whether or not a new or functional gene can help replace or inactivate a defective gene. Scientists have created two different types of gene therapy treatments based on which cell is being targeted. Somatic gene therapy involves the transfer of a section of DNA into any cell that does not produce sperm or eggs. However, germline gene therapy involves transferring a section of DNA into cells that produce sperm or egg. The effects of this therapy will be passed onto future generations; however, this is not a possibility with somatic gene therapy.

Subsequently, there are two main kinds of gene therapy techniques: gene augmentation and gene inhibition therapy. Gene augmentation therapy is a process in which the correct gene produces the necessary product of the defective gene, however, it does not replace the flawed gene in the DNA. On the other hand, gene inhibition therapy involves the replacement or inhibition of the flawed gene.

Recently, a group of researchers at the Netherlands Institute for Neuroscience and the Leiden University Medical Center have used gene therapy in order to treat nerve damage and regenerate nerve cells. The researchers combined gene therapy with a surgical repair procedure in order to stimulate the regeneration of the nerve cells and fibers. The results of the study show the impact that gene therapy can have in order to find new treatment options for people with nerve damage.

The nerve fibers in our bodies are very fragile and can be torn into pieces. Nerves in the neck can be torn out of place which can lead to paralysis. Currently, the only option that these people have is surgical repair, however, the success rate and implications of this technique are a major flaw. If done wrong, surgery can lead to further nerve damage and cell loss. Thus, researchers have been working for years to develop a new technique that is suitable and successful for any type of nerve damage.

With this new technique, many damaged nerve fibers and cells can be repaired at a faster rate than before. This development can help save many lives and treat many patients who are currently experiencing some sort of paralysis.

Subsequently, nerve regeneration or neuroregeneration can help treat many other diseases involving nerve cells and fibers. Currently, researchers are working on how gene therapy and neuroregeneration can help cure Alzheimer’s. Scientists from the Imperial College in London have prevented the development of Alzheimer’s in mice through the use of gene therapy. Alzheimer’s is a mentally destabilizing disease that impacts thousands of people across the world. With no proper cure, researchers are scrambling to find a new technique that can improve upon the current treatment options for Alzheimer’s. This new methodology created by the researchers in London can help cure Alzheimer’s through the implantation of a specific gene in the brain.

PGC1 is a gene that can inhibit the production of amyloid-beta peptide in the brain. In recent years, scientists have discovered that patients who have Alzheimer’s have accumulated a lot of amyloid-beta peptides in their brains. By inhibiting the production of amyloid-beta peptides, the PGC1 gene has the potential to cure Alzheimer’s and many other neurodegenerative disorders. Through gene therapy, the researchers can insert the PGC1 gene without replacing any other genes.

Though there are many hurdles to overcome, gene therapy is the future of the medical field. With the potential to cure many diseases ranging from psychedelic all the way to neurodegenerative, genetic therapy is a more reliable treatment option for many diseases and can help save many lives.

However, genetic therapy has many implications that need to be discussed. Gene therapy can damage the human gene pool and can lead to unanticipated results. If any error occurs during the genetic introduction process, the patients can have a greater error in their genetics than what was expected. Subsequently, genetic therapy is not very cheap. Currently, it costs around one million dollars per treatment.

At the same time, there are many ethical and spiritual issues with gene therapy. Having the ability to change the genetic makeup of a human is very dangerous. The concern over human perfection has led to many debates and court cases. Imagine a world where parents could manipulate the physical and mental characteristics of their children. This would create a whole new social class where access was exclusive only to the elite who have the financial means to pay for this procedure.

Gene therapy has many pros and cons and it is ultimately up to the government and medical officials to decide on the true impact that it can have on our society. By restricting genetic manipulation, the government would allow researchers to help discover new techniques in order to cure many diseases, however, this would stop the creation of the “perfect” child. Until this decision is made, there will continue to be heated debates about the future of gene therapy.


1. Loukia Katsouri, Yau M. Lim, Katrin Blondrath, Ioanna Eleftheriadou, Laura Lombardero, Amy M. Birch. PPARγ-coactivator-1α gene transfer reduces neuronal loss and amyloid-β generation by reducing β-secretase in an Alzheimer’s disease model. Proceedings of the National Academy of Sciences. 2019.

2. Rivat C, Santilli G, Gaspar HB. Gene therapy for primary immunodeficiencies. Human Genetic Therapy. 2012.

3. Ruben Eggers, Fred de Winter, Stefan A Hoyng, Rob C Hoeben. Timed GDNF gene therapy using an immune-evasive gene switch promotes long-distance axon regeneration. Brain. 2019.


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