By Grant Jacobs 30/07/2017

Gene editing could be a big deal. It offers some great stuff. For a few applications it also raises some interesting ethical questions.

We might be able to treat some genetic disorders by convert a “broken” gene associated back to the working version of the gene, potentially providing a one-time, life-long fix for the patient. Gene therapy has a surprisingly long history and several cases of ‘gene therapy’ has been done.

Some medical conditions have genes being used when they ought not to be. Gene editing could shut off meddlesome genes that aren’t supposed to be active. We might also develop ways to target cells with the re-arranged genes associated with some types of cancer.

Edited butterfly wing patterns, from Barrangou & Doudna, Nature Biotechnology 34, 933–941 (2016).

The list goes on with many other applications, including in agriculture and elsewhere: gene editing is a general-purpose approach that can be put to a lot uses.

One application with thorny ethical question is editing embryos.

There’s also hype and over-blown claims – far too much of it.

Before you pitch yourself at ‘the media’, it’s not all their fault. There’s “the usual” mess from some quarters online that seem to come with any new development in biology. There’s also specialist speaking because their field. For example, some of this fuss comes from so-called futurists and transhumanists. Their view of international regulation and how society might develop can (sometimes) be interesting, but often their grasp of biology isn’t really good enough (to be polite).

Gene editing has recently been in the international media spotlight with news that an USA-based team successfully edited human embryos. This is not the first time human embryos have been genetically edited – for example, there are earlier efforts by Chinese groups who ended their work concluding that for them the edits occurred too rarely, and when they occurred too often had off-target edits or left a mixture of edited and non-edited cells.

News reports say the USA team found they could edit the cells with a high(er) rate of successful editing of their intended gene, with few off-target edits.

Their science has not yet been published. Reporting work to media before the research paper is published is not a good practice in my opinion. It can enable uncritical presentation of work because no-one can check the work itself until after the media fuss has died down.

The embryos that were edited were never intended to be grown to term and were destroyed.

This work is then very much a science-for-scientists study—it doesn’t “do” anything in the sense of being used on a person or applied as a therapy. It also doesn’t mean routine human embryo editing is just around the corner. It is another reminder that gene editing is increasingly becoming a technique we might “do stuff” with.

I hope I can bring you a few articles about gene editing and gene therapy over the coming few months.

I am writing from Asia, living off the rent of my house. Currently I’m based in one location (in Laos) rather than travelling in the hope is that I can try tackle a few longer science communication projects, among other things. These projects are being done at my expense.* My posts may not be as regular as I would like them to be as I will want to give priority to my longer-term projects.

If you’ve got particular questions you’d like addressed, or topics you’d like covered, let me know in the comments. First-time comments are moderated to filter out spam.

There are also related, but different, techniques which are easily confused. I’ve deliberately left these out here to avoid being confusing, but I’m happy to cover these two. For example, gene therapy doesn’t necessarily involve gene editing – it can be done using modified viruses.


* I would like to finance some of it by writing a few pieces for magazines or similar, but I have to admit I am feeling pretty despondent about (lack of) support for scientists wanting to write about science in New Zealand recently – a topic for another post, perhaps.

Featured image

Originally from ShutterStock, royalty-free images; sourced from The Conversation.

Related posts on Code for life

Kumara are transgenic

Haemophilia — towards a cure using genetic engineering

Human gene editing recommendations from USA science panel

Gene editing and GMOs in NZ, part three (there’s links to parts one and two at the start)

Food and genetic modification: better informed policy and legislation wanted

0 Responses to “Fixing our genes”

  • Hi Grant. Do you imagine that it will be possible to gene-edit the carrier mothers of X linked Ocular Albinism (Nettleship-Falls syndrome), and so prevent their future sons from being born with Ocular Albinism? Also, might it be possible to edit children and adult males already exhibiting ocular albinism?

  • Hi Chris,

    Thanks for your question. I have to admit I wasn’t expecting questions about specific conditions, which was a bit short-sighted of me! (No pun intended.)

    What I might do to is to write about what gene therapy in general needs, and perhaps weave an answer (of sorts) to your question in that.

    You probably already know that other ocular conditions have been tackled with gene therapy. One example is the treatment of Leber congenital amaurosis (LCA), first tried in 2007/2008. It didn’t bring 20/20 vision, but the treatment gave some gain of vision. Impressive to be able to do something, really.