Genetic Engineering Reduces Tau Level in Alzheimer’s Patients

The Genetic engineering strategy has been used by researchers to dramatically reduce the levels of tau in an animal model of the condition. Tau is a crucial protein that accumulates and tangles in the brain during the development of Alzheimer’s disease. The results from the investigators of Sangamo Therapeutics Inc. and Massachusetts General Hospital (MGH) could become a potentially promising treatment for patients having this illness.

Science Advances describes this strategy to involve a Genetic regulation technology called zinc finger protein transcription factors (ZFP-TFs). These are DNA-binding proteins that can be harnessed to target and affect the expression of specified genes. In this research, the therapy designed was aimed at targeting and silencing the gene expression codes for tau.

The Alzheimer’s affected mice received one shot of the injection for their treatment. The injection employed a harmless virus to deliver the ZFP-TFs to cells–directly into the brain’s hippocampus region. This treatment reduced the tau protein levels in their brains by 50% to 80% out to the longest time point studied, 11 months.

The most important part of the therapy was that it reversed a few of the animals’ brain cells’ Alzheimer-related damage. Senior author Bradley Hyman, MD, Ph.D., said, “The Genetic technology worked just the way we had hoped–reducing tau substantially for as long as we looked, causing no side effects that we could see even over many, many months, and improving the pathological changes in the brains of the animals. This suggests a plan forward to try to help patients.”