What’s in the biomedical pipeline for CRISPR gene editing?

Credit: Gaia
Credit: Gaia

One great attraction of Crispr, says Niren Murthy, a bioengineer at Berkeley, is that it could be a one-shot affair. You have the treatment and the gene is fixed for good, rather than you having to return to the doctor every few months. 

Some researchers hope that Crispr can combat cancer. One approach would use gene-editing to boost our immune system so that it is better at destroying tumour cells. Such cancer immunotherapy is already showing great promise, but “Crispr could make it more efficient or effective,” says [CRISPR co-creator Jennifer] Doudna. “The basic concept is to edit a patient’s T-cells [a type of white blood cell central to the immune response] and reintroduce them to the bloodstream so that they can recognise and attack cancer cells.”

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Atherosclerosis (a cause of stroke and heart disease) is another important target. Some people have a genetic vulnerability to it because their cells produce too much of a protein called PCSK9, which stops a molecule called LDL cholesterol from being broken down… That makes PCSK9-related atherosclerosis an ideal target for Crispr therapy. 

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Although Murthy says that widespread clinical use of Crispr therapies is still five to 10 years down the line, Doudna admits to being “constantly amazed at how quickly Crispr genome-editing has been adopted by researchers around the world”.

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