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Gene therapy breakthrough

USA – Making gene therapy treatment cheaper and faster to deliver is on the horizon, thanks to a breakthrough by scientists at Scripps Research.

The important development centres around a non toxic molecule called caraphenol A, which is ‘closely related to resveratrol, a natural compound produced by grapes and other plants and found in red wine’. It can be used to reduce the number of steps it takes to transfer ‘healthy genes into the patients’ blood stem cells’.

“If you can repair blood stem cells with a single gene delivery treatment, rather than multiple treatments over the course of many days, you can reduce the clinical time and expense, which removes some of the limitations of this type of approach,” said Bruce Torbett, PhD, associate professor in the Department of Immunology and Microbiology, who led the research, published in the journal Blood,

The team is now studying what makes stem cells so resistant to genetic modification, as it seeks to further improve efficiency and cost for a therapy often used to treat diseases that affect children.

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