Last month doctors from Great Ormond Street Hospital apparently cured a baby girl’s terminal leukaemia using an experimental gene-editing therapy. Does this breakthrough mark the beginning of an exciting new era for gene therapy, or is this simply another flash in the pan?

Are we on the brink of a gene-editing therapy revolution for cancer?

Yes 60.9 %

No 34.8 %

Additional comments:

“RNA-editing process is involved in the cancer already. Why do we need to interfere more?”

There was a very clear response to this opinion poll! There appear’s to be a strong feeling, at least among FLG readers, that we are now moving into a new era for gene-editing therapies, at least for cancer. Back at the turn of the millennium the future of gene therapy was far from certain, dogged by high-profile failures and dismissed by many simply as hype. We’re not the only ones asking this question. In the wake of Great Ormond Street Hospital’s success many commentators emerged to ask whether the technology had come of age, and was now ready to shake off it’s questionable past and move into mainstream medicine. 

One of the big game-changers, of course, is CRISPR. Developing potential therapies is now more efficient than ever, and CRISPR cures gene therapy of one of its major flaws by reducing the risk of ‘off-target’, adverse effects. The precision that CRISPR brings to gene therapy massively reduces the risk of cancer that emerged during early gene therapy trials in the early 2000s.

The major barrier to implementing gene therapies is delivery, an area that we explored during our coverage of Layla’s case last month. Very simply, gene therapy can be applied ex vivo, using genetically altered cells grown outside the body and then injected into the patient, or in vivo, through a direct infusion or injection of therapeutically altered DNA. At present gene therapy remains largely confined to the ex vivo world, through manipulations made to easily accessible systems like blood. During in vivo therapy the editing vectors become much harder to control. Will enough cells be altered to have a therapeutic effect? And will those edits be made in the right parts of the body?

But even with these concerns, it is clear that we are entering a new era for gene therapy, one being greeted with confident optimism.


This poll is in collaboration with Source BioScience, an international provider of laboratory products and services.

source bioscience


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