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Many neurological disorders are genetically inherited, and for a large number of these diseases we know the exact mutations which cause the disease. Yet, development of an effective treatment remains challenging. Zooming in on a fault in the genetic code with a magic eraser, to directly correct the disease-causing error, has been made possible with CRISPR-based gene editing, which has great potential as a treatment to inherited disorders. But the technology is not without its challenges. 

A young researcher at DTU has the ambition to produce molecular tools which will make it possible to develop more effective, selective, and thus safer, genetic medicines, in a quicker and more efficient manner than ever before.

CRISPR gene editing has been used as a research tool in laboratories for some years, but the risk of so-called ‘off target effects’ means that rigorous safety testing is necessary, to assess, in particular, whether the treatment might act unintentionally in different tissues throughout the body. In many cases, genetic diseases only affect one tissue, so sending the magic eraser directly to those cells and ensuring that it doesn’t reach others could be highly beneficial.

Lisa Riedmayr is establishing a research group at DTU’s Bioengineering Department, with the help of a Lundbeck Foundation Fellowship. Her work is focused on developing novel tools for precision genetic medicine. Initially, Lisa Riedmayr will direct her efforts towards Developmental and Epileptic Encephalopathy (DEE) which is a rare and debilitating inherited disorder that affects children. With proof-of-concept of her lab’s approach secured, Lisa Riedmayr is confident that this fellowship will support her in the future development of therapies for a range of genetic disorders.

“I’m very excited to get started; to make an impact on genetic medicines. My aim is that genetic medicines will be available for many different mutations and diseases one day soon. With this fellowship I’m able to get one step closer to that goal.”

Authorization and clinical implementation of CRISPR-based medicines have been challenges by the risk of “off-target effects”, particularly regarding the potential for genetic changes in reproductive cells of a patients’ body, which could later affect future generations. By ensuring that the molecular machinery can recognise the cell type that it’s in, and can work only in the cells of interest, Lisa Riedmayr aims to show that CRISPR can be developed as safe, effective and reliable treatments. Using a screening tool that she has built, Lisa Riedmayr and her team will scan the genetic code in search of patterns which will enable the gene-editing enzymes to recognise and target the disease- and neuron-specific genes of interest.

“I’m dreaming big, in the hope to be able to disseminate findings that CRISPR therapies can be recognized by the authorities as safe therapeutic strategies, so that processes can be optimised for the benefit of patients, using tools that make its development faster and more effective. Germline editing is something that the medicine agencies raises as a concern, and my research has the potential to completely eradicate this risk.”