Gene therapy involves the introduction of healthy genes into the patient's cells that instructs the body to build proteins to cure the disease.
Genes are the basic units of heredity. Researchers have constantly been trying over the years to implement gene therapy for the treatment of various conditions. As a result, gene therapy has been successful in certain disorders which are caused due to a single well-known fault in a particular gene. In such cases, gene therapy is carried out by introducing a healthy gene into the patient’s cells. The cell utilizes the newly introduced healthy gene to make healthy proteins that reduce symptoms or cure disease. Parkinsonism refers to a condition of the nervous system involving the progressive destruction of brain cells resulting in movement-related complications, stiffness of the body, and shaking of arms and legs. Various clinical trials have been implemented to facilitate gene therapy in treating parkinsonism.
Parkinson’s disease is a disorder of the nervous system which affects the body parts controlled by nerves and is caused due to the dysfunction or damage of nerve cells inside the brain that produces dopamine. Dopamine is a chemical messenger that transfers signals within the brain's nerve cells and from the brain cells to other parts of the body. Dopamine plays a significant role in regulating a person’s movements, memory, learning, motivation, sleep, and emotional responses. The major symptoms seen in patients with Parkinson’s disease are trembling hands and legs, slow movements, stiffness in the body, loss of balance, and decreased ability to perform actions like smiling, talking, blinking, and writing.
Parkinsonism is a generalized term used to indicate a group of nerve-related disorders with symptoms similar to Parkinson’s disease, like slow movements, trembling hands and legs, body stiffness, and loss of balance. Parkinsonism includes several nerve-related disorders with similar symptoms, out of which Parkinson’s disease is the most commonly affected condition. The major difference between Parkinson’s disease and parkinsonism is that Parkinson’s disease is caused due to damage or destruction of the nerve cells in the brain, whereas parkinsonism can be caused by various factors like severe head injuries, side effects of certain medications, toxins, and any nerve-related conditions.
Genes are the basic units of heredity. They carry unique genetic information often used in the cells to produce new proteins. Genes play a major role in determining the physical appearance of a person. They are responsible for the inheritance of mental and physical traits of individuals from their parents and help pass the traits from one generation to the next in a family. Sometimes, genes also play a role in developing a particular disease or condition. If a person inherits a defective gene, he may have the possibility to develop a range of diseases.
Gene therapy is one of the latest advancements in medicine, which refers to treating certain diseases with the help of healthy genes instead of medicines. Gene therapy can be performed in several ways based on the type of disease. The most common gene therapy method is by introducing a healthy gene into the patient’s cell, which would instruct the body to produce the missing proteins needed for organ functioning. Another form of gene therapy involves the replacement of a defective gene with a copy of a healthy gene. Gene therapy can also be done by deactivating a gene that does not function properly.
Researchers believe that gene therapy can be useful in treating parkinsonism based on the fact that this therapy can prevent the damage and death of the dopamine-producing cells in the brain. Researchers point out the benefit of this therapy over the other conventional treatment measures, as gene therapy can slow or stop the disease, whereas conventional treatment methods can only reduce the symptoms of the disease. The current research on gene therapy and parkinsonism aims to deliver healthy genes directly into the affected nerve cells of the brain.
Gene therapy uses a carrier agent called a vector to deliver healthy genes into the brain cells to treat parkinsonism. Genetically modified viruses are the commonly used vectors to deliver therapeutic genes to the brain. These viruses are modified in such a way that it does not cause any infection and does not spread to parts other than the targeted area of repair. Each viral particle will enter only one cell (area of target) and introduce the therapeutic genetic material. Even though the virus disappears after delivering the genetic material, the therapeutic effect of the genetic material remains in the cell of the patient for a very long time. Adeno-associated viruses and lentiviruses are the commonly used vectors in delivering therapeutic genes to Parkinson's patients.
Many clinical trials have been going on to implement various outcomes of gene therapy in the treatment of parkinsonism. They are as follows:
Even though gene therapy has many advantages over conventional treatment methods, it is still under clinical development. However, gene therapy has shown great efficiency in the management of parkinsonism. In addition, the side effects of the drugs used in treating parkinsonism and the reduced potential of medications to treat the disease at severe stages have made gene therapy a reliable option to treat parkinsonism. Hence, with more clinical trials and better advancements, gene therapy stands as a hope for the new generation of clinically relevant treatment options for parkinsonism.
Last reviewed at:
30 Jan 2023 - 5 min read
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