Cellular and Molecular Neurotherapy Laboratory

The lab's flagship project is to develop a treatment for Huntington's disease using gene therapy and a technology known as RNA interference. This rare hereditary disease usually appears after the age of 40 and is manifested in uncontrolled movements and changes in personality. At present, there is no treatment that can inhibit the progression of the disease and patients are offered only palliative care.

Huntington's disease is caused by the mutation of a specific gene. This generates an abnormal protein which destroys the nervous cells situated in the striatum, the part of the brain responsible for controlling movement. “Our idea is to block the progress of this mutated gene. To do this, we plan to use RNA interference, small RNA sequences which bind themselves to the mutated gene and deactivate it, which will prevent the production of the abnormal protein”, explains Professor Déglon.

But even once the area to be treated has been identified, the RNAs still need to penetrate the targeted cells. To do this, Prof. Déglon’s team wants to use a viral vector, a virus that has been rendered safe and whose content and envelope have been modified to allow it to transport RNA molecules to the required area. “The RNA molecules will then absorb themselves into the targeted cells and act by correcting the pathological effects caused by defective genes” explains Prof. Déglon. “This treatment requires only one procedure, after which the patient's own cells create the treatment locally and continuously, inside the patient's own brain”.

But before such a treatment can be developed, a number of measures must be taken, from improving understanding of the disease and brain function, to clinical validation of the treatment. Situated at the heart of the CHUV, the Molecular and Cellular Neurotherapy Lab combines the know-how of different medical professionals, from biologists to neurologists to neurosurgeons, which is crucial to linking each of these different steps.

“We regularly meet the neurologists and neurosurgeons in the department as part of our day-to-day business.” says Prof. Déglon, “Their expertise is essential to developing effective patient treatment protocol. In the case of Huntington's disease, the neurosurgeons will be in charge of injecting the viral vector into a very specific part of the patient's brain. That is why it is so important that they be present throughout the development of the study to validate it. This interaction is vital to ensuring the treatment’s success.”