New Alzheimer’s Disease Treatment Using Knock-in Mice: Latest Alzheimer’s News


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Australian genetic research company, Ozgene, together with Curtin University, have published the results of a groundbreaking new study on Alzheimer’s. Using their knock-in mouse models, a likely cause of the progressive cognitive illness has been discovered. This could lead to new prevention and treatment opportunities for Alzheimer’s disease. 

READ –  Latest Alzheimer’s Treatment Using Knock in Mice  [plus podcast audio March 2022] 

What is Alzheimer’s Disease? 

Alzheimer’s disease is a progressive, irreversible brain disease in which nerve cell deterioration gradually affects thinking and memory.  

This condition generally manifests slowly and progressively gets worse over time, becoming severe enough to limit daily function.  

The early onset Alzheimer’s disease can happen as young as 40 or 50 years of age but typically the risk of developing the disease increases after 65 years.  

As the global population continues to age, the number of people with Alzheimer’s continues to rise as well.  

It is estimated that nearly 10 million people worldwide currently suffer from Alzheimer’s – and by 2050 that figure could reach as high as 160 million. Despite the vast number of people affected by Alzheimer’s, the progress toward finding a cure or treatment has been slow.  

Is Alzheimer’s the same as Dementia? 

Dementia is an umbrella term that refers to loss of memory, thinking and communication skills severe enough to interfere with daily life.  

Alzheimer’s disease accounts for 60-70 percent of dementia cases. The differences between Alzheimer’s and Dementia are mostly in the types of symptoms presented.  

The main difference lies in whether or not cognitive impairment affects day-to-day living, which separates them into groups: 

  • Alzheimer’s disease – where the cognitive impairments affect everyday living.
  • Vascular dementia – where the cognitive impairments do not affect everyday living.
  • Dementia with Lewy bodies – where there are fluctuations in cognition that vary between periods of clarity and confusion, but ultimately do not affect your ability to carry out normal everyday tasks.

New Alzheimer’s discovery from Australia 

A groundbreaking new Australian study has discovered a possible cause of Alzheimer’s disease, with potentially significant new prevention and treatment possibilities for Alzheimer’s.  

The research was looking into the progressive accumulation of toxic protein deposits between neurons, called plaques, which are a well-known characteristic of Alzheimer’s disease.  

These plaques are made of amyloid beta, which is a waste product of the amyloid precursor protein (APP). Until now, researchers have been unsure where the amyloid beta comes from or why it deposits in the brain. 

The new Alzheimer’s study published in PloS Biology found that the amyloid beta most likely originates from fat-carrying particles in the blood, called lipoproteins.  

This ‘blood-to-brain pathway’ is crucial since lowering lipoprotein-amyloid levels in the blood and preventing their entry into the brain may lead to new treatments for Alzheimer’s disease, such as drugs that target lipoprotein amyloid or simply by introducing dietary changes.  

This discovery has resulted from years of research by a collaborative group of Australian scientists from Curtin University, led by Professor John Mamo, and Ozgene Pty Ltd, a leading provider of mouse models for scientific research.   

The cornerstone of this study was data obtained utilizing a custom designed mouse model of Alzheimer’s. 

Why are mice used for Alzheimer’s? 

Mouse models allow researchers to study genetic, environmental, and behavioral elements of Alzheimer’s disease as well as test drug candidates before human testing. Mice are often used in Alzheimer’s research because their brains are similar to ours.  

They have the same types of neurons, which connect in roughly the same way, and both mice and humans naturally produce amyloid beta proteins with age, which can lead to cognitive decline.  

Genetically unmanipulated wild-type mice could potentially be used to show amyloid plaque formation in mice. The mouse amyloid, however, is not identical to humans and does not lead to the formation of the plaque in the same way as in humans.  

Therefore, the application of wild-type mice as a research method for Alzheimer’s disease is limited. 

Why are transgenic or knock-in mice used for Alzheimer’s? 

Genetically modified mice are the most widely used animals to study Alzheimer’s disease (AD).  

These animal models are typically transgenic or knock-in mice that are genetically engineered so that their brains age faster, similar to how human brains age in AD patients.  

Transgenic mice can reproduce many features of human disease, including age-related pathology. The identification of Alzheimer’s disease genes has allowed transgenic modeling to be developed that reproduces many important features of the disease. 

Transgenic mouse models of Alzheimer’s disease typically overexpress either proteins linked to Familial AD (FAD), mutant Amyloid Precursor Protein (APP) and Presenilin (PS) or Tau that can cause an early-onset Alzheimer’s.  

More recently developed App knock-in mice introduce clinical mutations in the mouse App gene. Many of these recent mutant mice are humanized mice, that is, they are genetically manipulated to produce large amounts of human amyloid beta in the brain.  

The humanized AD mouse models show significant plaque formation in the brain cells that builds up and eventually destroys the cells. This leads to deterioration in memory and cognitive abilities. 

Unique Alzheimer’s knock-in mice generated 

The Australian research team found the existing knock-in and humanized AD mouse models pathologically interesting, but not particularly useful. 

 Since these mice already had an exaggerated human amyloid expression in the brain, they could not be used to explore whether the amyloid can travel from the blood to the brain.  

Therefore, Prof. Mamo’s team needed a novel mouse model to prove their hypothesis of a blood-to-brain pathway for Alzheimer’s disease. 

A unique mouse model of Alzheimer’s disease was created for the study. These Ozgene knock-in mice were different from other Alzheimer’s disease models because they were genetically engineered to produce human lipoprotein amyloid specifically in the liver, not in the brain.  

As the research team predicted, the mice suffered inflammation in the brain, accelerated brain cell death and memory loss, which confirmed their blood-to-brain pathway theory. 

Clinical trial for Alzheimer’s disease 

This study demonstrated that the presence of amyloid proteins in the blood is related to higher chances of developing Alzheimer’s disease. There are some medications that could specifically target lipoprotein amyloid, reducing a person’s risk or slowing the progression of Alzheimer’s disease, which opens up possibilities for exciting new research. Professor Mamo’s team is currently conducting a clinical trial in Australia using a drug called Probucol.  

It is based on prior research showing that this historical cardiovascular medication lowers lipoprotein amyloid production and improves cognitive function in mice. In time, this drug could bring relief to millions of people and improve their quality of life. 


Knock-in Mice FAQ 

What are transgenic mice? 

Transgenic mice have a transgene from another source introduced into their genome, expressing it in “trans”. The altered genome in transgenic mice can help gain understanding into a cellular process or protein function, as well as to develop new functions that could be used to study physiological responses. The disadvantage of transgenic mice is that there is little control over spatial and temporal expression levels since the transgene is randomly integrated into the genome, not inserted in a specific locus. 

What are knock-in mice? 

Knock-in mice are produced by inserting reporter genes, human genes, transgenes or point mutations in a specific mouse locus to create genetically modified mice for medical research. The difference between the knock-in and knockout methods is that knock-in mouse models are utilized to induce the expression of a transgene or gene mutation, whereas knockout mice attempt to stop or reduce the expression of a particular gene. 

What are humanized mice? 

Humanized mice can be described as knock-in mice expressing human genes and proteins. The goal of humanized mice is to mimic human protein and disease function in a mouse model. Humanized mouse models have become an important pre-clinical tool for biomedical research and they are widely used to study and develop treatments for human diseases, such as Alzheimer’s disease. 

Latest Preclinical Study and Data on Alzheimer’s and Knock-in Mice 

Here is the very latest scientific data and study paper on Alzheimer’s knock-in mice.  Published June 2022 

New Alzheimer’s Disease Treatment Using Knock-in Mice Summary 

Ozgene’s participation in the possible Alzheimer’s disease treatment is an exciting discovery because it confirms that knock-in mice are at the forefront of genetic research.   

The custom designed knock-in mice were pivotal for identifying a likely cause of Alzheimer’s, leading the way to a clinical drug trial. 

If you wish to contact Ozgene in connection with their mouse models or Alzheimer’s disease research using knock-in mice, please enquire here: 

Indianapolis, IN, USA 

Ph: +1 248 762 3626 


Ozgene Pty Ltd (Headquarters) 

PO Box 1128 

Bentley DC, WA 6983 


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