Motor neuron disease (MND) is an untreatable and rapidly progressive fatal condition. In order to develop treatments to slow down, stop and ideally repair the damage, we must first understand precisely what is going wrong and where.
Starting with the 'what'; recent studies confirm that problems are occurring with the messages that make protein in cells. This leads to protein misplacement and malfunction.
Until now we have been probing this system with blunt tools but our current array of primary and collaborative laboratory techniques allow us to precisely understand what is going wrong. This is like comparing an old map to a new GPS that can tell you the position of an object in a room of a house on the street you are looking for.
As for the ‘where’, we may instinctively think that the main problem in motor neuron disease lies within the motor nerves. Although this seems logical, we now know from careful studies that the disease is not that simple and that actually, the supportive cells called glia (not nerve cells) may well be the primary culprits.
Resolving these issues will allow rationally engineered and targeted therapies to treat this devastating condition.
The ideal technology for this project is human induced pluripotent stem cells (hiPSCs), which allow the generation of theoretically unlimited numbers of the MND patients very own motor nerves and glia in a petri-dish.
The Patani Lab is a group of scientists based at the Institute of Neurology which is part of University College London and we use motor neurons that we grow from patient-derived stem cells to study what happens in motor neuron disease.
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