To understand gene therapy, it is important to first understand what a gene is and how it works. Genes are small portions of DNA which form the basic functional units of heredity (U.S. National Library of Medicine, 2020). Genes play a very important role in determining our physical and mental traits. Some genes can be used as instructions to make specific proteins within the cell that perform different functions. However, sometimes random chance events can result in gene mutation, causing proteins to malfunction.
Currently, many research studies are looking into how gene therapy can treat neurodegenerative diseases such as Parkinson’s disease. Gene therapy offers a new method for treating and preventing various conditions, instead of using traditional drugs that we use today. Different ways in which gene therapy can be used to treat diseases is by replacing, silencing, or correcting faulty genes (Axelsen & Woldbye, 2018). One example of gene therapy includes inserting a healthy gene into the cells of a patient with the goal of treating that condition. The new gene that is inserted into cells can instruct the body to build new proteins that are absent but are required for normal functioning of organs (European Parkinson’s Disease Association, 2020). Another example of gene therapy is inserting a gene into the body to replace another gene that is non-functioning. This could repair and prevent cells from potentially dying (European Parkinson’s Disease Association, 2020).
Although Parkinson’s Disease is not usually inherited, it is believed that eventually, gene therapy will be able to prevent the death of cells in the early stages of the disease. Gene therapy could be life changing for those with Parkinson’s disease because currently, treatments are only made to control their symptoms, whereas gene therapy could potentially halt or slow the progression of Parkinson’s disease (Leigh, 2019). In addition, current medications that are available are usually only effective during the early stages of the disease and it becomes less effective as the patient develops more intrusive symptoms (Leigh, 2019).
The hope of current research is to be able to insert genes directly into the neurons of the affected areas of the brain. Some trials are in process to determine whether gene therapy could instruct brain cells that are not damaged by Parkinson’s disease to produce dopamine in order to recover the lost function that resulted from the loss of dopamine-producing neurons. Other research is looking into how gene therapy could prevent the nerve cells in the basal ganglia of the brain from becoming overactive. When the basal ganglia is overactive, it can affect the movement of individuals (European Parkinson’s Disease Association, 2020). Growth factors are also being used in research trials, in which researchers are using them to promote the rejuvenation and survival of neurons. In previous studies, introducing growth factors prevented the loss of neurons in the substantia nigra, located in the midbrain, which improved function in rhesus monkeys with experimentally induced Parkinson’s disease (Tuszynski, 2002).
Although gene therapy is not yet available as a treatment option for Parkinson’s disease, there are currently ongoing clinical trials that will hopefully obtain approval from regulators in the near future. Once these gene therapy options are approved, this treatment will be more widely available for the public.
References
Axelsen, T., & Woldbye, D. (2018). Gene therapy for Parkinson's disease, an update. Retrieved February 11, 2021, from https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6027861/
European Parkinson's Disease Association. (2020). Gene therapy. Retrieved February 11, 2021, from https://www.epda.eu.com/living-well/therapies/surgical-treatments/gene-therapy/#:~:text=Gene%20therapy%20and%20Parkinson's&text=Gene%20therapy%20has%20the%20potential,affected%20area%20of%20the%20brain
Leigh, S. (2019, March 20). Gene therapy shows initial promise for Parkinson's disease. Retrieved February 11, 2021, from https://www.ucsf.edu/news/2019/03/413696/gene-therapy-shows-initial-promise-parkinsons-disease
Tuszynski, M. H. (2002, May 1). Growth-factor gene therapy for neurodegenerative disorders. Retrieved February 11, 2021, from https://pubmed.ncbi.nlm.nih.gov/12849545/
U.S. National Library of Medicine. (2020, September 01). What is a gene? Retrieved February 11, 2021, from https://medlineplus.gov/about/general/genetics/newhome/