Discovery without boundaries
Discovery science is a key area of focus for the MRC; between 60 and 70 per cent of the MRC’s research portfolio is classified as discovery research. The MRC is committed to expediting the impact of discovery research by bringing it closer to users in industry and clinical medicine. In addition to the many researcher-generated discovery science programmes, focal areas of discovery research funded by the MRC include:
The Discovery for Medicine topics reflect the MRC’s strategic assessment of the opportunity to develop the necessary knowledge-base in these areas. The MRC’s commitment to funding discovery research, across the breadth and depth of the entire MRC portfolio, is continued through a focus within this sub-set of topics. The following case studies and quantitative data demonstrate the impact of the MRC's strong track record in leading scientific discovery.
Zinc finger proteins discovered by MRC-funded research are used in first gene-editing trial in humans
MRC-funded research from Sir Aaron Klug’s lab at the MRC Laboratory of Molecular Biology underpins a historic gene editing trial run by a US biotechnology company. Scientists are attempting to edit a gene inside a body for the first time, to permanently change a person’s DNA to cure a disease. In a ground-breaking clinical trial, researchers in California have treated a patient with gene editing tools to treat a severe inherited disease. The patient, who has Hunter syndrome, was treated in November 2017 with a specifically engineered zinc finger nuclease to target a deficient gene in his liver.
MRC-funded scientists gain licence to edit genes in human embryos in the UK
In February 2016, Dr Kathy Niakan’s team at the Francis Crick Institute became the first to receive permission from the Human Fertilisation and Embryology Authority (HFEA) to use the genome editing technique 'CRISPR-Cas9' in human embryos. Dr Niakan was subsequently listed as one of Time Magazine’s Top 100 Most Influential People of 2016.
Scientists develop technique to control proteins inside live cells to treat disease
In 2015, scientists at the MRC Laboratory of Molecular Biology developed a new technique using ‘designer molecules’ to control proteins inside living cells. Named BOLT, this technique could allow scientists to control defective proteins involved in a wide range of diseases.
Scientists partner with industry to develop new diagnostic test for inherited heart disease
In January 2015, scientists at the MRC London Institute of Medical Sciences published the results of a meticulous ‘proof of concept’ study looking for a mechanism underlying inherited heart disease. By carrying out a comprehensive analysis of the titin gene, the team identified specific defects which are important for causing dilated cardiomyopathy. This work led to collaboration with Illumina Incorporated to develop TruSight, a new diagnostic test for inherited cardiac conditions which was launched in May 2015.