Alzheimer's Disease May Be Prevented by Gene Regulation Therapy

Novel gene regulation approach treats brain diseases such as Alzheimer's in a laboratory using zinc finger proteins as per research at the DZNE (Germany), at Massachusetts General Hospital (USA), and at the genomic medicine company Sangamo Therapeutics, Inc., published in the journal Science Advances.

The zinc finger proteins specifically bind to the DNA that code for the protein Tau without altering it. This helps in reducing the Tau production in the brain and thereby preventing nerve damage.

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‘Gene regulation therapy could protect against Alzheimer's disease by providing a sustained Tau protein reduction in the brain with a single-dose administration. This demonstrates that zinc finger protein transcription factors (ZFP-TFs) can reduce Tau mRNA and proteins in neurons both acutely and long-term in the adult brain, thereby placing the technology as a starting point for future therapies and clinical research in humans.’

The Tau protein drives the development of certain degenerative brain diseases called tauopathies where Tau accumulation within nerve cells destroys synapses and cell death.

Alzheimer's disease (AD) is a neurodegenerative disease that leads to gradual memory loss and behavioral changes. It is characterized by the formation of beta-amyloid plaques and the tau proteins in the brain tissues, long before the actual symptoms occur.

Zinc Finger Protein Therapy

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The study designed a gene regulation therapy using zinc finger protein transcription factors (ZFP-TFs) that provides sustained Tau protein reduction in the brain when administered to a preclinical mouse model of Alzheimer's disease – one of the most studied tauopathies.

The ZFP-TFs induce long-term changes in Tau expression without altering the genetic material of the cells in the process, unlike gene-editing approaches, which cut or change the DNA itself.

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It was observed that a single intravenous or intracranial administration of tau-targeted ZFP-TFs resulted in a 50 to 80% decrease in tau levels by 11 months (the longest time point studied) with no obvious side effects. This may idealize the generation of new treatments for tauopathies such as Alzheimer's disease and frontotemporal dementia.

"Under certain conditions, such as Alzheimer's disease, Tau proteins can have the effect of a cell toxin. Thus, there is an ongoing effort to reduce their amount in the brain. Nerve cells normally produce Tau proteins. With the help of zinc finger protein technology zinc finger protein technology, we have now been able to achieve a long-lasting reduction of Tau production, and thereby significantly reduce the toxic effects of the plaques," says Susanne Wegmann, head of a research group at the DZNE - German Center For Neurodegenerative Diseases in Berlin.

The study thus demonstrates that ZFP-TFs can reduce Tau mRNA and proteins in neurons both acutely and long-term in the adult brain. This places the zinc finger technology as a starting point for future therapies and clinical research in humans.

"These compelling results published in Science Advances demonstrate the potential of our highly specific and efficient zinc finger protein transcription factor technology for. There are currently no disease-modifying therapies approved for patients with Alzheimer's disease or other tauopathies. This publication represents the promise of Sangamo's zinc finger protein transcription factor approach to potentially provide a one-time treatment to slow or stop disease progression", says Bryan Zeitler, Director of Gene Regulation at Sangamo.

Source-Medindia