Medical Research Council - London Institute of Medical Sciences

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Spheres of Influence
06 March 2013

Spheres of Influence

Although the idea of curing diseases by replacing faulty genes with healthy ones is decades old, the revolutionary potential of genetic therapy has yet to be unlocked. Any practical therapy would have to overcome the multiple challenges of inserting healthy genes into the correct tissue and targeting only malfunctioning cells, while ensuring that no harmful immune response follows. Present-day treatments tested in trials consist of injecting patients with a harmless virus loaded with the replacement gene, which the virus then splices into the host cells’ DNA. But with our immune systems honed to kill viruses, the procedure can be risky. Researchers working on alternatives have produced protein-based pellets (pictured) loaded with genetic material for delivery inside diseased cells. The nanoscale-sized pellets should be friendlier to the immune system than viruses, and could potentially also be shaped into rods, spheres or coils, to help them enter only targeted tissue.

Written by Tristan Farrow

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