Medical Research Council - London Institute of Medical Sciences

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A Swell Treatment

Using organoids – mini lab-grown tissues – to demonstrate success of gene editing

02 September 2021

A Swell Treatment

Like old lubricant glooped over time into sticky residue jamming a bicycle chain, sticky mucus around organs of the body can cause real problems. This is what happens in people with cystic fibrosis – one of the most common genetic conditions, and one without a cure. Researchers in search of treatments trialled a new version of a well-known gene editing technique on organoids – miniature artificial organ structures. Using this ‘prime editing’, they replaced the faulty section of DNA with healthy sequences in cells with cystic fibrosis. Healthy organoids swell (right) when a substance called forskolin is added, but those with the cystic fibrosis mutation do not because of a fault in a molecular channel that causes the mucus build up. Following treatment, organoids showed the swelling behaviour, illustrating that the gene editing was successful and safe, and swelling hopes that these approaches could eventually be used to help patients.

Written by Anthony Lewis

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