What to Know About Gene Therapy for Alzheimer’s Disease?

Though promising, there are still many obstacles to overcome in the field of gene therapy for Alzheimer's. It's one of several newly developed treatments that might aid in symptom reduction or illness progression.

Alzheimer's disease is a neurological ailment that progresses over time and is characterised by memory loss, cognitive decline, and behavioural changes. The symptoms of Alzheimer's can only be somewhat relieved by the medicines that are currently available, despite decades of research.

Emerging medicines, however, hold new promise in light of recent important advances in genetics and medicine. One such treatment that goes after the underlying genetic and molecular causes of Alzheimer's is gene therapy.

This article will describe the mechanism of action of gene therapy in the treatment of Alzheimer's disease and how it compares to other available and potential future therapies.

What is gene therapy, and how does it work?

By altering your DNA, gene therapy is a medical strategy that helps treat inherited and acquired disorders. It entails adding, deleting, or changing particular genes to treat a disease's underlying causes.

Gene therapy transfers beneficial genes into your body through non-viral or viral vectors. The desired genes are delivered to the target cells using these vectors. These target cells are commonly found in the brain in Alzheimer's disease.

The inserted genes integrate into your DNA once they've reached the intended cells, where they start the production of particular proteins or compounds.

Such proteins can:
  •  beta-amyloid in Alzheimer’s
  • promote cell survival
  • correct genetic anomalies
  • reduce harmful protein accumulations like
  • modulate immune responses

How can gene therapy help people with Alzheimer’s disease?

Alzheimer's disease gene therapy is currently in the experimental stages. several human investigations as well as several animal research have been conducted.

It is unclear whether Alzheimer's disease is inherited or brought on by a specific gene. However, some inherited genes could make you more susceptible. Alzheimer's may also be caused by genetic changes in your cells, though it's unknown whether these changes are inherited or brought on by environmental factors.

Your brain's neurons are affected by these gene changes because they change the way that proteins function. The goal of gene therapy for Alzheimer's disease is to transfer changed genes to help restore the protein activity that has been disrupted.

Trials of human gene therapy for Alzheimer's have successfully and safely delivered genes that encode a protein that encourages neuronal development. In a 2020 experiment on animals, the amount of amyloid plaque in mice's brains was successfully reduced, and the mice's memory was enhanced.

Although preclinical investigations have yielded encouraging outcomes, clinical studies have been less clear, warn researchers.

Can gene therapy cure Alzheimer’s?

Although gene therapy has promise as a treatment, it is currently difficult to use it to treat Alzheimer's. This is because Alzheimer's is a complex disorder with numerous underlying causes.

With the use of the gene-editing method CRISPR, researchers can accurately change and edit genes to fix mutations linked to diseases like Alzheimer's. However, environmental and lifestyle variables also play a role in the onset of Alzheimer's, making its eradication difficult.

Gene therapy for Alzheimer's attempts to: rather than offering a permanent cure:
  • reduce symptoms
  • slow progression
  • delay onset
Continuous research is necessary to improve gene therapy techniques and boost their efficiency in controlling the symptoms and progression of Alzheimer's disease.

What other promising new Alzheimer's treatments are there?

Another treatment option being looked into by researchers is a potential cure for Alzheimer's disease. promising new therapies for Alzheimer's disease include:
  • Monoclonal antibodies: Acuranumab is one of several monoclonal antibody treatments that specifically target beta-amyloid plaques in the brain to delay cognitive loss.
  • Tau protein targeting: A crucial component of what causes Alzheimer's is being managed by therapies that concentrate on lowering aberrant tau protein tangles.
  • Anti-inflammatory drugs: Drugs that target inflammation in the brain are being researched by scientists. These medicines might be able to slow the progression and symptoms of Alzheimer's.
  • Stem cell therapies: Approaches using stem cells are designed to repair or replace lost neurons in Alzheimer's patients' brains.
  • Neuroprotective agents: To improve cognitive performance and stop the course of the disease, researchers are looking into drugs that support healthy neural function.
  • Vaccination strategies: To increase the immune system's defence against proteins linked to Alzheimer's disease, researchers are looking into vaccines that target the beta-amyloid or tau proteins.
  • Precision medicine: A 2023 study found that individualised treatments based on a person's genetic and molecular profile are becoming more common in Alzheimer's care.
  • Neurotrophic factors: To cure Alzheimer's, scientists are investigating medicines that promote the growth and survival of neurons.
  • Combination therapies: The effectiveness of combining various treatments to effectively control Alzheimer's disease is being investigated in several trials.

It's crucial to keep in mind that numerous of these treatments are still undergoing various stages of clinical trials to assess their long-term effectiveness and safety.

What is the most promising treatment for Alzheimer’s?

Disease-modifying medications are three of the most effective Alzheimer's treatments, according to the Alzheimer's Society:
  • donanemab
  • lecanemab (Leqembi)
  • remternetug
Lecanemab was authorised by the Food and Drug Administration (FDA) in 2023. It was the first new Alzheimer's medication to be approved in 20 years. Clinical trials are still ongoing for the other two.

What are current treatments for Alzheimer’s?

There are already several medicines that can help manage symptoms and delay the advancement of the disease, even though gene therapy is still a relatively new kind of care for Alzheimer's. They consist of:
  • Cholinesterase inhibitors: Medication is frequently prescribed by doctors to treat Alzheimer's patient's memory loss and cognitive decline by elevating neurotransmitter levels. 
These include:
  • donepezil (Aricept)
  • rivastigmine (Exelon)
  • galantamine (Razadyne)
  • Memantine: Memantine (Namenda) controls glutamate activity, which is involved in learning and memory, to treat moderate to severe Alzheimer's symptoms.
  • Behavioural and psychiatric medications: To treat mood and behavioural symptoms like depression, anxiety, and agitation, doctors may prescribe antidepressants and antipsychotic drugs.
  • Lifestyle modifications: Regular physical activity, a balanced diet, social interaction, and mental stimulation can all assist in enhancing general well-being and may even prevent the course of Alzheimer's.
  • Supportive therapies: You can manage your everyday duties and keep your independence with the aid of occupational, speech, and physical therapy.

These therapies don't offer an Alzheimer's disease cure, but they can reduce some symptoms and enhance quality of life.

The goal of ongoing research is to create better treatments and ultimately discover a means of stopping or curing Alzheimer's.


Although there is currently no known treatment for Alzheimer's, gene therapy has the potential to both reduce the disease's course and enhance cognitive function in those who already have the condition.

The precise manipulation of genetic and molecular variables that contribute to Alzheimer's disease is known as gene therapy. It can restore injured neurons and even target beta-amyloid plaques, lessen inflammation, stop the loss of brain cells, and diminish beta-amyloid plaques.

The development of an Alzheimer's gene therapy treatment is still in its preliminary stages. To confirm its safety and effectiveness, additional research is required.


Can you live 20 years with Alzheimer's?

Some people live as long as 20 years with the disease.

Is Alzheimer's a slow death?

Alzheimer's disease tends to develop slowly and gradually worsen over several years.

Is Alzheimer's disease fast or slow?

Alzheimer's disease tends to develop slowly

Is Alzheimer's genetic?

In most cases, Alzheimer's does not have a single genetic cause.

Does Alzheimer's run in families?

In most cases, Alzheimer's does not have a single genetic cause.

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