Science Questions

How can we prevent the undesirable effects of gene therapy?

Sat, 29th Oct 2011

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Question

Ian Borukhovich, Facebook asked:

Have methods to anticipate undesirable effects of a new therapy been improved since previous failed attempts? Has legal regulation of new therapies been enhanced over the past decade or so?

I have heard of many promising and almost unbelievable successes resulting from stem cell therapy and I think that such therapy has a big role in the future treatment of physiologic malfunction.

Answer

We posed this question to Simon Waddington and Adrian Thrasher from University College London...

Simon -   Gene therapy and stem cell therapy researchers have spent a huge amount of time not only trying to make the most efficient gene therapy vectors in stem cell treatments but also to ensure that they are as safe as possible.  And when were using different gene therapy vectors for example, if were using ones that are based on HIV these actually insert themselves into the genome and they can therefore disrupt the genome.  So there's certainly been work over the past 8 years to try to firstly make them as safe as possible so they don't affect the surrounding genes.  But also, as Kathy High alluded to in her presentation today, what weve been doing for many years in gene therapies actually is providing a supplementary copy of the gene, but actually, still leaving the mutated gene in.  Whereas what Kathy is doing, and various other researchers in the community, is creating tool kits to be able to actually repair the gene and return it to what we would perceive to be its normal function.  And that, although its inefficient at the moment is very exciting as almost an ideal safe gene therapy.

Ben -   And as with all the types of medicine, there must be all sorts of regulation, all sorts of boxes that you need to tick before you can actually put something on the market, or even before you can get it into trials in people.

Adrian Thrasher -   Of course, gene therapy is no different from any other pharmaceutical therapy in terms of its regulation.  We obviously have to go through a fairly rigorous ethical process and also be able to ensure that we can give medicines that are made or manufactured in the right way that's safe for people.

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