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RNA interference is a powerful technique for silencing genes and, therefore, has therapeutic potential. However, although the technique works well in cells, it has proved difficult to make it work in specific diseased tissues of a living animal — particularly the brain, in which the blood–brain barrier (BBB) often blocks entry of therapeutic agents.
Reasoning that neurotropic viruses penetrate the BBB, a multi-institutional team created a hybrid molecule that included a peptide of the rabies virus attached to a lentivirus that contained RNA sequences designed to silence specific genes. The technique worked in single cells. An intravenous infusion of the hybrid molecule in mice penetrated the BBB, attached to the acetylcholine receptor on neu…