Parkinson’s Gene Therapy – 2020 Treatment

Updated on - Written by
Medically reviewed by Kimberly Langdon, MD

parkinson's gene therapy

Parkinson’s disease has been around for years and until now, no one has an absolute answer to how nerve cells dead, and how they lost the ability to produce dopamine. Some studies[1] correlate Parkinson’s aging or changes in genes that lead to exposure to hazardous chemicals that can interfere with the production of dopamine from the nerve.

There is good news on the horizon for people suffering from Parkinson’s disease. A recent study[1] has shown that the use of the most advanced technology used gene therapy may help improve symptoms of Parkinson’s disease, such as stiffness, tremors, and other problems with motor function, helping with the safety and tolerability of a patient.

Parkinson’s disease (PD)[2] is the second neurodegenerative disorder most common throughout the world after Alzheimer’s disease. PD involves the malfunction and death of nerve cells in the half part of the brain known as the substantia nigra. These parts are in charge of controlling movement and coordination. The cells then produce a chemical called dopamine, a neurotransmitter that sends messages to these parts of the brain. As Parkinson’s disease progresses, the amount of dopamine decreases, resulting in motor symptoms like tremor, rigidity, slow movement, and difficulty with balance.   

Parkinson’s Diagnosis And Current Treatments 

parkinson's gene therapy

What if someday a neurologist could say reassuring a worried patient, “Do not worry, it’s just Parkinson”. Perhaps it will be the case as a research[3] and clinical trials give us better treatments and perhaps even a cure. 

The current treatment landscape may alleviate some of that excitement. The long-term effects of L-Dopa[4] are uncertain and may lead to serious adverse reactions. Despite the introduction of dozens of other alternative medicines and therapies such as DBS (deep brain stimulation), only partially eliminating the symptoms and not stopping progression, L-Dopa still presents as the most effective drug treatment. However, when you look at some specific areas to study the possibilities for the future still seem quite favorable.

Parkinson’s Diagnosis

Parkinson’s disease is difficult to diagnose because there is no one specific test for the condition. Symptoms vary from person to person and several others have symptoms similar to diseases, which means misdiagnosis may occur. Trials have been made to improve the early diagnosis of Parkinson’s disease[5].

Early treatment is based on early diagnosis, it is important to note the first symptoms[6] of Parkinson’s disease and consult a doctor as soon as possible. 

Your doctor may recommend these following tests:

Blood test[7]

This help can for other conditions, such as abnormal thyroid hormone levels or liver damage.

MRI or CT scan

A scan may detect signs of a brain tumor or stroke. If there are no signs of a brain tumor or stroke, most MRI or CT scans of patients with Parkinson’s disease appear normal. A person with a normal scan of the brain, but the symptoms of PD can have PD.

Positron emission tomography (PET)[8]

This is a test image that can sometimes detect low levels of dopamine in the brain. PET scans are costly and some hospitals do not offer them, so this option is not regularly available.

Single-photon emission computed tomography (SPECT)[9]

This is also a dopamine transporter (DAT) scan.

Whatever the results of the examination, the doctor will consider mainly the signs and symptoms of the person making a diagnosis.

Parkinson’s Treatment

Although there is no popular Treatment For Parkinson’s Disease, some current options can help control symptoms and improve the patient’s quality of life. Various treatments are consisting of medication, physical therapy, and Natural Remedies For Parkinson’s Patients. These trials include:


Dopamine precursor: a drug that can pass through to the brain cells and easily convert to dopamine. Help in managing Parkinson’s disease.

Catechol-O-methyltransferase (COMT) inhibitors: inhibited the action of catechol-O-methyltransferase enzyme that involves in degrading neurotransmitters.

Dopamine agonists[10]: dopamine-producing receptor is activated and helps in managing the disease.

MAO-B inhibitors: increases the amount of dopamine-producing in the basal ganglia by inhibiting the activity of an enzyme that breaks down dopamine.

Deep brain stimulation[11]: A surgical procedure to improve motor symptoms such as stiffness, slow movements, tremor, rigidity, and trouble walking.


Carbidopa / Levodopa enteral suspension: The drug transmits to the small intestine through a stomach tube through a keyhole performed through surgery.

Thalamotomy[12]: The destruction of part of the thalamus to help alleviate movement disorders.

Pallidotomy: pallidotomy is the destruction of the globus pallidus, one part of the brain responsible for the symptoms of Parkinson’s disease.


Physiotherapy: It can help to balance and improve motor mobility problems.

Occupational therapy: Helps to update the daily activities of these foods to eat, read, and dress patients.

Parkinson’s Gene Therapy

A gene therapy for Parkinson, a viral vector delivers a new functional gene can helps cells to create enzymes or neurotransmitters. Together with existing Parkinson’s disease drugs, it can create a much stronger result.

Novel approaches based on gene therapy for Parkinson is not a cure. However, it has several potentials as it allows more effective management to control symptoms longer. Typically, gene therapy introduces the gene into the cells to make proteins or enzymes which are key to the treatment of the disease.

What is gene therapy?

Gene therapy[13] for Parkinson involves stimulating the cells to produce proteins that normally they do not. Professionals carry out the therapy by using a genetically engineered virus that has all the disease-causing components removed. This allows us to use the efficacy of a viral vector delivery system. Viruses are very good at infecting cells.  

Can gene therapy be applied as a Parkinson’s treatment?

There is a group of several clinical trials[14] that approach various gene therapy strategies for treating Parkinson’s disease, including:

Glutamic acid decarboxylase

GAD[15] increases a brain chemical production associated gamma-aminobutyric acid or GABA. Gene therapy for GAD can set into the subthalamic nucleus in the brains of PD patients. The objective is to increase the presence of GABA in the brain, helping to rewire the abnormal brain of Parkinson’s.

Aromatic amino acid decarboxylase

AADC[16] is an important enzyme in the conversion of levodopa to dopamine and the decline of AADC in the brain can be used for changes in the effectiveness of treatment of long-term levodopa. Gene therapy for AADC was preferable in the putamen in the brain. This therapy aim at increasing the amount of AADC, making more effective treatment.

Neurturin (NTN), glial-derived neurotrophic factor (GDNF)

A protein that is inside GDNF family of nerve growth factors. Specialists can apply a neurturin gene in the several brain regions in PD patients to support the survival of neurons.


Tyrosine hydroxylase, guanosine triphosphate cyclohydrolase, and aromatic amino acid decarboxylase are three important enzymes in the synthesis of dopamine. This therapy has been introduced into the putamen of PD patients to increase the production of dopamine.

How does gene therapy work in the brain?

parkinson's gene therapy

A virus, or a vector is a small lump of nucleic acid that penetrates a cell and hijack the machinery of the cell. This then replicates its nucleic acid and produce viral proteins, which frequently cause disease in the process. Certain viruses are still able to incorporate their genetic material into the genetic material of the cell they infect. The scientists intelligently take advantage of these processes to insert therapeutic genes. 

The harmful DNA pieces are removed from the virus and the gene of choice is incorporated into the viral structure. Then, the modified virus is injected into a specific part of the body, thereby allowing the cells to “spread”.  If everything goes according to plan, those pieces of DNA are used by the cell by the cellular machinery to produce the protein of choice.


The question was whether gene therapy could cure genetic diseases. After obtaining information about Parkinson’s gene therapy, gene therapy has the potential to become a Parkinson’s disease treatment. However, it cannot be an official approval as an effective treatment yet.

However, to perform the efficacy of Parkinson’s treatment, gene therapy must go through several clinical trials. Hopefully, these tests can provide treatment that will become a regular procedure in medical practices for genetic diseases like PD.

Frequently Asked Questions

What is Parkinson’s disease?

It is a chronic neurodegenerative disorder that affects movement, symptoms of the common disease include tremor of the limbs, even at rest, in addition to the stiffness of the arms, legs, and trunk, slowness of movement, and impaired balance and posture.

Is there a specific test to diagnose?

There is no definitive standard test that marks Parkinson’s disease. Instead, a neurologist will perform several neurological tests and physical examinations to rule out other diseases that mimic the side effects of Parkinson before concluding PD.

Can Parkinson’s Be Prevent?

To date, there’s no known way to prevent Parkinson’s disease. But, there are several treatment options, including drug therapy and/or surgery can reduce symptoms and make living with the disease easier.

Is Parkinson’s disease contagious?

Parkinson’s is not a contagious disease, there’s no need to worry about being in close contact with someone who has it.

Can Parkinson’s disease be inherited?

Although most cases of the disease are not the result of an inherited gene, there is evidence that this could be a possibility in some people.

What is gene therapy?

Gene therapy for Parkinson is a treatment category that involves the introduction of DNA into cells to modify the proteins that are created and thereby improve symptoms or even cure the disease.

Why are viruses used in gene therapy?

Viruses are used in gene replacement therapy due to their natural ability to enter the body’s cells. In gene replacement therapy, scientists rework or adapt a virus so that it can be used as a vehicle or delivery vector, without causing disease in humans.

What are the risks of gene replacement therapy?

Most of the risk of gene replacement therapy has to do with the viral vector used to deliver new genes into cells. Long-term risks are unknown. The general risks listed unwanted immune system reaction, Unable to turn off the new working gene, the new gene inserts in the wrong place. The wrong cells may be targeted.

+ 16 sources

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  3. Feng, L.R., and Maguire-Zeiss, K.A. (2010). Gene Therapy in Parkinsonʼs Disease. CNS Drugs, 24(3), 177–192. Available from:
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  10. Parkinson’s UK. (2020). Parkinson’s drugs. Available from:
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  12. (2020). Thalamotomy for Parkinson’s Disease | Patients & Families. Available from:
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  14. Lin, J., Xie, C., Zhang, S., Yuan, W., and Liu, Z.-G. (2017). Current Experimental Studies of Gene Therapy in Parkinson’s Disease. Frontiers in Aging Neuroscience. Available from:
  15. LeWitt, P.A., Rezai, A.R., Leehey, M.A., Ojemann, S.G., et al. (2011). AAV2-GAD gene therapy for advanced Parkinson’s disease: a double-blind, sham-surgery controlled, randomized trial. The Lancet Neurology, 10(4), 309–319. Available from
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