How a Liver-Damaging Drug May Give a Boost to Gene Therapy
June 9, 2016
(Science Magazine) – Researchers who want to treat diseases by ferrying a gene into cells often face the hurdle of safely introducing the DNA into enough of them to make a difference. Now, scientists have come up with a novel way to make gene-modified cells in the liver take over much of that organ: They cripple the unmodified cells. This seemingly risky strategy, which relies on the liver’s exceptional regenerative skills, has passed its first test in mice. If equally successful in people, it could be a boon for treating many inherited diseases involving the liver.