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Wellcome Trust Research Round-Up: 09/02/2015

9 Feb, 2015

Our fortnightly round-up up of news from the Wellcome Trust community…

Vaccination combats severe diarrhoea in Malawi

malawi vaxResearch published in Lancet Infectious Diseases has found that the introduction of a rotavirus vaccine in Malawi has significantly reduced the number of hospital admissions of young children with severe diarrhoea.

Rotavirus is the leading cause of severe, acute gastroenteritis among infants and young children throughout the world and is responsible for an estimated 453,000 deaths among children under age five each year. To combat this, a vaccine for rotavirus was added to the schedule of immunisation routinely given to babies in Malawi by the WHO in 2012.

However, no evaluation of the vaccine in real-world use had been carried out. Now, a four year Wellcome Trust-supported programme that involved more than 1,400 children presenting with diarrhoea at the Queen Elizabeth Central Hospital, Malawi, has found that the rotavirus vaccine reduced the risk of severe diarrhoea by 64%, as well as the total community burden of rotavirus hospitalisation by 43%.

Dr Naor Bar-Zeev, who led the research team at the Malawi-Liverpool-Wellcome Trust Clinical Research Programme, said: “Our new data show that these efforts are now resulting in really positive outcomes for the health and wellbeing of children in Malawi. The success of this vaccine demonstrates the significant impact it can have on countries with a high burden of diarrhoeal disease, particularly when it is introduced into childhood immunisation programmes at the earliest stage possible.”

The next stage of the research will examine the duration of protection afforded by the vaccine and its effectiveness against different genetic forms of rotavirus.

Two and a half thousand women could benefit from mitochondrial donation in the UK

Alison and Niamh, Niamh lost her battle to mitochondrial disease

Alison and Niamh. Niamh lost her battle to mitochondrial disease aged four-and-a-half. Alison is co-founder of The Lily Foundation, a charity representing families suffering from mitochondrial disease.

Almost 2,500 women of child-bearing age in the UK are at risk of transmitting mitochondrial disease to their children, according to the most recent estimates published in the New England Journal of Medicine.

The research offers the most recent evidence yet of how many families could potentially be helped by new IVF techniques to prevent mitochondrial diseases. Mitochondrial diseases are caused by inherited mutations in the DNA contained in mitochondria – tiny structures present in every cell that generate energy. Mitochondrial diseases can be devastating and particularly affect tissues that have high energy demands – brain, muscle (including heart), liver and kidney.

New IVF-based techniques have been developed which have the potential to prevent the transmission of serious mitochondrial disease. Known as ‘mitochondrial donation’ the techniques involve removing faulty mitochondria inherited from the mother and replacing them with the healthy mitochondria of another woman. The nuclear DNA, containing 99.9% of genetic material from the mother and father, remains unchanged.

Researchers at the Wellcome Trust Centre for Mitochondrial Research at Newcastle University have now calculated how many women have disease-causing mutations in their mitochondrial DNA in order to estimate how many could potentially benefit.

They calculate that 2,473 women in the UK, and 12,423 women in the US, aged between 15 and 44 years, are at risk of passing on potentially lethal mitochondrial DNA disease to their children. This equates to an average of 152 births per year in the UK, and 778 births per year in the US.

On Tuesday 3rd February 2015, MPs in the House of Commons voted overwhelmingly in favour of a motion to allow further research on mitochondrial donation and eventual licensing of the technique by the HFEA.

Different patterns in “switch off” of genes in developing male and female brains in the womb

B0005909 BabyNew research published in the journal Genome Research has revealed differences in developing male and female brains in the womb.

The study focussed on epigenetic changes, or molecular “switches” that regulate the way that genes are activated. Researchers particularly focused on an epigenetic change called ‘DNA methylation’ to try and understand how differences in this process in male and female brain development may contribute to sex differences in behaviour, brain function and disease.

DNA methylation is the best understood epigenetic process: a chemical modification to one of the four bases that make up our genetic code. In this study significant changes in DNA methylation across brain development were found at more than 7% of the 400,000 genomic sites assessed.

Professor Jonathan Mill, of the University of Exeter Medical School and King’s College London, who led the study, said: “The prenatal period is a time of dramatic plasticity, when the brain is laying down the structures that control neurobiological function across life. Understanding the way in which genes are activated during this important period in the brain could teach us about the origins of disorders with a neurodevelopmental component, such as autism and schizophrenia.”

The study analysed over 200 brain tissue samples from the MRC/Wellcome Trust Human Developmental Biology Resource taken from foetuses 23 to 184 days after conception to find changes in the way that genes are regulated during human brain development

In other news…

Bill CastellSir William Castell, Chairman of the Wellcome Trust, was awarded the BioIndustry Association (BIA) Lifetime Achievement Award at the BIA’s Gala Dinner at the Natural History Museum.

‘Electricity’ supported by the Wellcome Trust has been nominated for best screenplay in the National Film Awards. Cast your vote now!

Syncona LLP, an independent subsidiary of the Wellcome Trust, has invested an extra £5 million in gene therapy start up NightstaRx Ltd (“Nightstar”) to treat choroideremia.

The Wellcome Trust Sanger Institute has announced a collaboration with AstraZeneca to exploit the full potential of innovative CRISPR technology in the field of drug discovery. CRISPR technology, sometimes known as ‘genetic scissors’, allows scientists to make precise changes to genes.

Image credits: Top: University of Liverpool, Middle: The Lily Foundation, Bottom:  Bill McConkey, Wellcome Images. Bill Castell – Wellcome Library

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