Developing neuroinflammation-directed precision AAV gene therapy for neurodegenerative diseases
Developing self-regulating gene therapy for neurodegenerative diseases
Neurodegenerative diseases, like Multiple sclerosis, Alzheimer’s and Parkinson’s disease, are a medical and societal problem. Millions of people suffer from these neurodegenerative diseases for which there is no cure. Gene therapy is in development for many neurodegenerative diseases. Harmless adeno-associated viruses can be used as transport vehicles to deliver a therapeutic gene to a patient. Although this virus mediated delivery by itself works well there is a major drawback, once a therapeutic gene is delivered, there is no control over therapeutic factor production by this gene. Continuous uncontrolled production of a therapeutic factor, even when it is no longer needed from a disease point of view, increases the changes of negative side effects.
In this project we will first search for human DNA-elements that can be used as inflammation sensitive on/off-switches to regulate the production of a therapeutic factor after the gene has been delivered to the patient. Next, we will test if identified inflammation sensitive gene switches indeed switch-on or off the production of a therapeutic factor in response to inflammation in an animal model for Multiple sclerosis. We expect that the therapeutic factor is only produced during the time, and only at the place where, in the nervous system inflammation occurs. Once an inflammation site has been cured by the therapeutic factor, the production should automatically be switched off.
Since, neuro-inflammation is a hall-mark of many neurodegenerative diseases we expect these inflammation sensitive gene switches can be used in gene therapies for many neurodegenerative diseases.
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