is director of the in Berlin, Germany, and the head of the Department of Regulation in Infection Biology at the Helmholtz Centre for Infection Research. Her work led to her co-discovery of the gene editing tool CRISPR Cas9, which she explains here.
What is the CRISPR gene-editing method?
CRISPR Cas9 is a mechanism that bacteria have evolved to defend themselves against viruses. It has now been harnessed to edit genomes in a large variety of cells and organisms. It can also be used to modify gene expression, and to study epigenetic marks.
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It’s cheap, easy to use, efficient and versatile, and the scientific community has adopted it very quickly.
Can you give us an example of how it might be used?
I would take the example of human genetic disorders for which the only treatment is really to correct the mutation of the gene that causes the disease.
With certain blood disorders, you might one day be able to collect the sick cells of the patient, correct the mutation, verify that nothing has gone wrong in terms of the manipulation of the genome and then re-implant those cells back in the patient and correct the disease.
But this is the ultimate goal. CRISPR already has a large beneficial effect in medicine because it helps us understand mechanisms behind disease.
You will be at an international summit in Washington DC this week to discuss gene editing. Do you think there should be a moratorium on editing the human germline – altering DNA that can be inherited?
There have been technologies before that could target specific gene sequences. So the idea of having a technology that would allow the manipulation of the human germline isn’t new.
But the previous technologies were less efficient. CRISPR is so easy to use that it has led to a number of developments for different purposes. It’s like any technology: there’s a good side to it and there is an ethical responsibility with regard to how to use it.
How do we decide how to use this technology?
Just by discussion. There is perhaps a misunderstanding by the public about what the technology does and how it works. It allows more precise genetic changes than all the breeding technologies that have been used before – so the organisms are genetically much cleaner.
I think all those around the table discussing the ethics – which includes scientists, clinicians, ethicists and the public – should first understand the technology and understand that it is a good technology, that it really allows us to accelerate the understanding of functions of genes. That will be important for the development of biotechnology and biomedicines.
Genetics is very important, the risk is the misuse of a technology, like for every technology, but I think that right now biologists want to use the technology for good reasons. Then after that, manipulation of the human germline needs to be discussed.
Is there ever a case for germline editing?
There would be reasons with regard to certain types of diseases. I hope that using the technology with the idea of changing human characteristics will not be pursued.
When it comes to using it for therapeutic and preventative purposes – not to change traits that could be inherited throughout the population – then the debate will be for certain kinds of diseases for which maybe the manipulation of the human germline will be considered. But then the question is whether society wants to go there. Philosophically and sociologically speaking, I have lots of issues with this.
Did you expect your work to cause this debate?
It was unexpected. The findings came a little bit all of a sudden. But it comes down to basic science – CRISPR is a very nice example of how basic science on an obscure immune system in bacteria can potentially lead to powerful technology.
You have said this is a democratic technology because it’s so cheap and easy to use. But you’re embroiled in a patent dispute over it
The technology doesn’t belong to the scientists, it belongs originally to the bacteria. But there is money involved, whether I like it or not. When there is a discovery in science there is always a patent, especially in the US. But everyone is using this technology very freely in the lab. All the material to make the technology work is available for free. It’s just if a biotechnology company wants to develop it commercially for a certain type of disease and there is a lot of investment involved. So that’s why it’s democratic – no one has to ask for access if anyone wants to use it on a research level in academia.
What do you hope will have come out of your research in 10 years?
I hope that technology will be developed to the point at which CRISPR Cas9 can be delivered in cells and tissue to treat severe human genetic disorders.
Emmanuelle Charpentier was speaking to a group of journalists at the in Berlin in November. This is an edited report of her comments
Image credit: Peter Steffen/AP/PA
