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Motor neurone signalling and the effects of RNA in MND

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Dr Pietro Fratta completed his first MRC-MND Association Clinical Research Training Fellowship in 2014. Last year he was awarded a new £1.16 million Clinician Scientist Fellowship to continue his research at University College London, studying the earliest physical changes that affect motor neurons in MND (our reference 946-795). Our contribution to this four year research fellowship is £280,000.

Pietro Fratta
Dr Pietro Fratta, University College London

As his first Fellowship progressed, Dr Fratta became more interested in the field of RNA biology, where he is rapidly establishing himself as an expert. His latest project aims to see whether RNA plays a pivotal role in the earliest signs of cellular damage that occur in MND.

RNA is the cell’s copy of our genetic material known as DNA; Dr Fratta is hoping to establish if the transport of RNA molecules along the nerve fibres is impaired and if so, whether there are particular versions of RNA that are particularly important for motor neurone health and survival.

Several lab studies have shown that the process of transporting things up and down the motor neurones is impaired long before the physical signs of damage are seen. His research will seek to find out what RNA molecules are present in both the cell body of the motor neuron and the nerve fibres.

Motor neurones are very long cells. When grown in a dish in the lab they grow in a tangled mess where the end of the neurone loops round and becomes close to the start of it. So if you want to find out where the RNA is along the neurone – at the start or at the end – the way they grow naturally makes this very difficult to work out. To resolve this Dr Fratta is using specially designed growing dishes called microfluidic chambers. The design of the dish forces the motor neurones to grow straight, making the study of RNA transport much easier. (Perhaps think of it like training runner beans to grow up poles in the garden).

The motor neurons will initially be obtained from a mouse model of familial MND” explains Dr Fratta.

We will then validate our findings using the latest stem cell techniques, where human motor neurons will be created from skin cells of patients with known gene mutations.”

For more information on funding research involving animals please see our website: www.mndassociation.org/animalresearch

Throughout June 2016 MND Awareness Month will be highlighting the rapid progression of the disease in its powerful Shortened Stories campaign, sharing the experiences of people currently living with MND, or who have lost loved ones to the disease, through art, poetry and film.