Gene therapy in Alzheimer's disease

Australian researchers have made a breakthrough in Alzheimer's research, demonstrating what they describe as the first gene therapy-based approach to treating advanced forms of the disease.


Through experiments in mice, they have been able to demonstrate how the activation of a key enzyme in the brain can prevent the type of memory loss associated with advanced forms of Alzheimer's and even reverse it.


The research was conducted at Macquarie University, where researchers Lars and Arne Ittner were investigating the role of a key enzyme in the brain called p38gamma. Through previous research, the researchers had shown that by activating this enzyme in mice with advanced dementia, they could modify a protein that prevents the development of Alzheimer's symptoms.

Based on this, the scientists conducted experiments on mice with advanced Alzheimer's disease to see not only how cognitive decline could be slowed, but also how the function of this protective enzyme could be restored to normal levels for even greater benefit.


The researchers discovered that by introducing genetic material, they could activate the p38gamma enzyme in a way that not only stopped the memory decline in the mice, but actively improved their memory despite the advanced nature of their disease.


The study detailing the discovery will be published in the journal Acta Neuropathologica in September.