Is a new dawn of widespread gene therapy on the horizon?


By Michael le Page It could be possible to target HIV hiding in immune cells Gene therapy – using genes to treat or prevent diseases – is already saving lives. But it’s still very much an experimental treatment used on a few individuals, rather than a routine therapy. CRISPR will help change that. For starters, it will be much cheaper and easier to develop treatments to the point where they are ready to test in animals and people. And these treatments should be safer. “It’s a new age in gene therapy,” says Oskar Ortiz of the German Research Centre for Environmental Health in Munich. Unlike germline engineering (see “Will CRISPR gene-editing technology lead to designer babies?“), changes made using gene therapy can’t be passed on to children. The conventional technique involves adding extra DNA to cells, but there is no way to control where it lands – and if it lands in the wrong place it occasionally results in cancer. With CRISPR gene editing, DNA can be added to a precise spot. Even then it can sometimes be added to the wrong place, but ways to minimise this have already been developed. What’s more, treating some diseases requires altering existing genes rather than adding new ones – and gene editing excels at this. If it proves safe, CRISPR could be used to modify cells in the body to treat a wide range of diseases. The tricky part will be delivering the gene-editing machinery and new DNA to cells inside the body. Fortunately,
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