Investigational Gene Regulation Therapy Lowers Levels of Tau Protein in the Brain and Could Protect Against Alzheimer's

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The Tau protein plays a key role in the development of certain degenerative brain diseases called tauopathies.

The Tau protein plays a key role in the development of certain degenerative brain diseases called tauopathies. Tau accumulates and aggregates within nerve cells, causing destruction of synapses and cell death. The consequence is a progressive dysfunction of cognitive, motor and psychological abilities.

Results of new research published in the journal Science Advances demonstrate that a gene regulation therapy using zinc finger protein transcription factors (ZFP-TFs) 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. A one-time intravenous or intracranial administration of tau-targeted ZFP-TFs decreased tau levels by 50 to 80% out to 11 months, the longest time point studied. The zinc finger technology was developed by Sangamo Therapeutics and has now been applied by the researchers to reduce Tau protein in the adult brain.It could be the starting point for a new generation of treatments for tauopathies such as Alzheimer's disease and frontotemporal dementia.

Read more at: DZNE - German Center for Neurodegenerative Diseases

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