Promising Results for Gene Silencing Therapy in Alzheimer's Disease

Cure for Alzheimer
pixabay Alzheimer's disease is one of the most common neurodegenerative diseases worldwide, and there is currently no cure. However, a new genetic therapy has been tested and shown promising results in reducing the levels of tau protein, one of the causes of Alzheimer's disease. 

The BIIB080 genetic therapy successfully reduced tau protein levels by up to 50% in 46 patients

Indicating that gene silencing approach using antisense oligonucleotide (ASO) called BIIB080/IONIS-MAPTRx could be a solution for Alzheimer's disease. By suppressing tau protein production at the genetic level, it is hoped to slow the progression of Alzheimer's disease.

The drug is safe and effective for mild Alzheimer's patients

All patients in the phase I clinical trial completed the treatment period, with more than 90% of patients completing the post-treatment period. Mild to moderate side effects occurred in patients who received the drug, but no serious adverse events were reported.

This genetic therapy is a new approach in treating Alzheimer's disease

Currently, there is no drug specifically targeting tau protein. Available drugs such as aducanumab and lecanemab still focus on reducing amyloid plaque accumulation.

Further research is needed to confirm the effectiveness of BIIB080 genetic therapy in larger and more diverse populations.

However, the phase I clinical trial results show significant progress in demonstrating that tau protein can be successfully targeted with gene silencing drugs to slow or even reverse the progression of Alzheimer's disease and other diseases caused by tau protein accumulation.

Overall, this new genetic therapy offers hope in the treatment of Alzheimer's disease. Further research is still needed to ensure the effectiveness and safety of this therapy. If this genetic therapy is successfully developed further, we may one day be able to reduce the burden of Alzheimer's disease on millions of people worldwide.

To learn more, please read the following journal:

Mummery, C.J., B√∂rjesson-Hanson, A., Blackburn, D.J. et al. Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer’s disease: a phase 1b, randomized, placebo-controlled trial. Nat Med (2023).


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