Medical Research Council - London Institute of Medical Sciences

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Protein Therapy
28 April 2018

Protein Therapy

Proteins are more than just a bodybuilder’s best friend. They are the building blocks of life, and how we make them is dictated by our DNA. Over the past few decades, gene editing technologies have helped us to tease apart and even replace mutated bits of DNA that cause genetic diseases. Researchers have recently developed a new tool called CasRx that applies these genetic editing principles to DNA’s close relative, RNA. Since RNA is key to the protein-making process and if faulty can also cause genetic diseases, scientists have used this new technology to correct a protein imbalance in the cells of a patient with dementia. This image shows CasRx (purple) at work targeting RNA in human cells. CasRx could be great new addition to the genetic engineering toolbox, and has the potential to pave the way for a whole new range of gene therapies.

Written by Gaëlle Coullon

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