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We must keep using CRISPR-Cas9 technology

Today we released a joint statement with other medical research funders in support of research using gene editing techniques such as CRISPR-Cas9 to advance our understanding of disease in preclinical research and to explore their potential for future therapeutic use. Here our Director of Science Programmes Dr Rob Buckle discusses the huge potential of gene editing, and why any attempt to use it in a therapeutic context must be the subject of the kind of intense and rigorous debate that the scientific community and UK regulatory system has demonstrated in the past.

Wooden blocks with DNA letters on them

(Image: Duncan Hull on Flickr under CC BY 2.0)

 

Being able to edit the human genome is not a new idea or capability. For decades researchers have been developing potential gene therapy techniques to correct missing or faulty DNA and restore healthy gene expression in cells.

More recently, techniques have been developed that can edit the genome in a much more efficient and targeted fashion, and one such example, CRISPR-Cas9, has accelerated the field to such an extent that researchers can now make precise edits to the genome in a relatively easy, speedy, precise and error-free way. Read more

Why I use human brain tissue in my research

There are more than 12,000 brains stored and ready for use by researchers in ten banks across the country ― and they’re easier to access than you might think. Here Dr L. Miguel Martins from the MRC Toxicology Unit explains what he gets out of working with brain tissue and provides some tips for researchers starting out.

Miguel and his team, Miguel is second from right.

Miguel (second from right) and his team.

 

I found out about the availability of deceased human brain tissue for my work because I have long-term collaborations ― since at least 2003 ― with colleagues at the UCL Institute of Neurology, which supports the Queen Square Brain Bank for Neurological Disorders, part of the UK Brain Banks Network.

My team’s research focuses on studying the genetics and cell signalling networks involved in Parkinson’s disease. I see using human brain tissue as being able to come full circle: Parkinson’s is a human disease which we investigate using animal models of the disease, and then validate in brain tissue donated by patients with Parkinson’s. Read more

From tool to therapy: a timeline of monoclonal antibody technology

They started out as a useful tool for studying the immune system in the lab and now they’re a family of drugs treating millions of patients, with global revenues of nearly $75 billion in 2013. MRC funding and researchers have been entwined with the monoclonal antibodies story from the very beginning. Forty years ago this month, Nature published a paper by César Milstein and Georges Köhler which described how they’d made mouse monoclonal antibodies. Here we look at the landmarks on the 40-year journey.

They can fight disease, determine blood types, and diagnose pregnancy in minutes. Such varied uses, but the usefulness of monoclonal antibodies actually lies in their uniformity.

Antibodies are proteins that recognise and fight foreign invaders, such as bacteria or viruses. Monoclonal antibodies are tailored in the lab to recognise specific desirable targets, such as a marker on a cancer cell or a pregnancy hormone. They are then churned out in their identical multitudes, ready to become a drug, a diagnostic test, or a probe to study disease in the lab. Read more