Targeted gene editing

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The big problem with current gene therapy technologies is that the new genes insert at poorly controlled locations in the gene sequence. This has obvious problems and two subjects who were treated for the X-SCID immune deficiency developed leukemia, likely as a result of the therapy. This isn't exactly a great advertisement for gene therapy.

Now, Urnov et al. report that they have found a way to create targeted modifications. I can't get at the whole article, but based on the New Scientist writeup, what it looks like is that they've built "zinc finger nucleases" (ZFNs), which are amino acid/zinc complexes that will bind to specific base sequences, ligate them, and splice in the correct sequence. They get 18% replacement, which is apparently pretty good.

The experiment being described was performed in vitro. The researchers extracted blood and treated it externally. It's not clear to me if this will work in in vivo. Conventional gene therapy often uses a virus. I'm not sure I see how to use this technique outside of the test tube.

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