Early Use of Gene Therapy May Stop Plaques of Alzheimer’s Disease

MedicalResearch.com Interview with:

Dr. Magdalena Sastre PhD Faculty of Medicine, Department of Medicine Senior Lecturer Imperial College London

Dr. Magdalena Sastre

Dr. Magdalena Sastre PhD
Faculty of Medicine, Department of Medicine
Senior Lecturer
Imperial College London

MedicalResearch.com: What is the background for this study?

Response: Alzheimer’s disease is the most common neurodegenerative disorder, affecting over 45 million people around the world. Currently, there are no therapies to cure or stop the progression of the disease. Here, we have developed a gene therapy approach whereby we delivered a factor called PGC-1α, which regulates the expression of genes involved in metabolism, inflammation and oxidative stress in the brain of transgenic mice. This factor is also involved in the regulation of energy in the cells, because it controls the genesis of mitochondria and in the generation of amyloid-β, the main component of the neuritic plaques present in the brains of Alzheimer’s disease patients.

We have found that the animals with Alzheimer’s pathology treated with PGC-1α develop less amyloid plaques in the brain, perform memory tasks as well as healthy mice and do not have neuronal loss in the brain areas affected by the disease.

MedicalResearch.com: What are the main findings?

Response: The main take away message is that Alzheimer’s disease can be stopped by this type of gene therapy treatment if it is applied at early stages of the disease. We need to test if this kind of therapy could be used safely and effectively in humans, although there is some hope because similar kind of gene therapy approaches has had successful outcomes in other diseases, such as Parkinson’s disease.

MedicalResearch.com: What should readers take away from your report?

Response: I think we should potentiate gene therapy approaches in the future, making them less invasive, because currently the vectors need to be injected directly in the brain. There are new viral vectors that are being developed, capable of getting inside the brain using delivery routes that are less invasive.

We could also investigate in more detail potential drugs that affect the formation of PGC-1α, such as resveratrol and nicotinamide riboside, among others.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: It is very important that research in Alzheimer’s disease is funded, not only for translational investigations, but also on the basic mechanisms causing the disease, because this would help to find a more specific and effective cure.

MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.


Katsouri L., Lim YM, Blondrath K, Eleftheriadou I, Lombardero L., Birch AM, Mirzaei N, Irvine EE, Mazarakis ND and Sastre M. PPARγ-coactivator-1α gene transfer reduces neuronal loss and amyloid-β generation by reducing β-secretase in an Alzheimer’s disease model. PNAS (in press) (2016).

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Last Updated on October 12, 2016 by Marie Benz MD FAAD