UK scientists apply for licence to edit genes in human embryos

[Hamilcar]

http://www.nature.com/news/uk-scientists-apply-for-licence-to-edit-genes-in-human-embryos-1.18394


UK scientists apply for licence to edit genes in human embryos
Team from London's Francis Crick Institute wants permit to use CRISPR/Cas9 technology in basic research.

Daniel Cressey, Alison Abbott& Heidi Ledford
18 September 2015 Updated: 18 September 2015



Kathy Niakan, a researcher affiliated with the Francis Crick Institute, London's new £700-million (US$1.1-billion) biomedical-research centre, said on 18 September that she is proposing to use gene editing to provide "fundamental insights into early human development". In a statement released through the Crick, Niakan said that her team wanted to use technology based on the CRISPR/Cas9 system — a recently developed technique for precisely editing genomes that has become hugely popular in the biology community. Her application was first reported by The Guardian newspaper.

Editing the genomes of human embryos for a therapeutic use — for example, to eradicate a genetic disease — is illegal in the United Kingdom, but research work is possible under licence from the Human Fertilisation and Embryology Authority (HFEA). The body, which regulates fertility treatment and embryo research, has confirmed that it has received its first application for a gene-editing licence using CRISPR/Cas9. "It will be considered in due course," the HFEA said.


Nature special: CRISPR — the good, the bad and the unknown
In April this year, Nature revealed that a Chinese team had, for the first time, reported using the CRISPR/Cas9 technique to edit the genomes of human embryos. The work, led by Junjiu Huang, a gene-function researcher at Sun Yat-sen University in Guangzhou, involved attempts to modify the gene underlying the blood disorder β-thalassaemia. The research used non-viable embryos that could not result in a live birth, but nonetheless caused huge controversy.

Robin Lovell-Badge, a developmental biologist at the Francis Crick Institute, emphasized to Nature that whereas Huang and colleague's paper explored the correction of a genetic defect that would lead to disease, Niakan's work proposes asking more basic questions about human-embryo development. "Kathy has no intention of making changes to the genome for clinical application," he says.

Lovell-Badge says that China has guidelines on gene-editing work in embryos, "but these are not quite laws, and there would only have been local ethics committee approval". By contrast, if Niakan's application were given a license, it would mark "the first approval by a national regulator," he says.

Regulatory debate
Huang's team's April report spawned a flurry of scientific and policy meetings and statements as governments and policy experts wrestled with how or whether to draw the line on gene editing in human embryos.


Chinese scientists genetically modify human embryos
Shortly after the work was published, the US National Institutes of Health reaffirmed its ban on funding gene-editing research in human embryos — a ban that would likely also apply to non-viable embryos, it said.

But on 2 September, five UK research organizations — including the Wellcome Trust and the Medical Research Council — issued a statement urging the continued use of CRISPR/Cas9 in research, even in human embryos when ethically justifiable and legal.

A week later, a network of stem-cell researchers, bioethicists and policy experts called the Hinxton Group, said that after meetings in Manchester, UK, they had concluded that research involving genome editing in human embryos has "tremendous value to basic research".

And on 14 September, Britain's Royal Society and the Chinese Academy of Sciences announced that they would join forces with the US National Academy of Sciences and the US National Academy of Medicine to host a summit in December on germline editing (genetic changes to embryos, sperm and eggs).

Closely watched
The HFEA application is likely to be closely watched by researchers around the world, because the regulator has a global reputation for careful but progressive regulation of human-embryo work.

Sarah Chan, a bioethics researcher at the University of Edinburgh, UK, says there is confusion around what is permitted in different parts of the world regarding human-genome editing. Good regulation, she says, can help to make clear to scientists what they can and cannot do. "Because of its history of successful regulation, the UK could serve as a model for other countries," she says.

"While I am certain that people in other countries will be paying close attention to both how the HFEA handle this licence application and, if it is granted, how the research progresses, it does not really warrant this attention," says Lovell-Badge. "The use of genome-editing techniques in this context is really the same as using any other method on an embryo that is not going to be implanted into a woman, and which will be destroyed after a few days of culture".

Nature doi:10.1038/nature.2015.18394

[Syt]

And so it begins.

[Tamas]

Finally

[Fate]

And so it begins.

No.

More like, less of this:

(Child with cystic fibrosis)

[Tonitrus]

Assuming the research/application progresses in a regulated, transparent environment, what really is the downside to genetic engineering?  Humans will become "too good"?  Genetic diseases being eliminated is a bad thing?

If, say, we could eliminate every future occurrence of down syndrome, how is that bad?  Sure it would be bad if we used that capability to argue that we should eliminate living people with DS as well...but no one (sane) is arguing for that.

[Malthus]

Assuming the research/application progresses in a regulated, transparent environment, what really is the downside to genetic engineering?  Humans will become "too good"?  Genetic diseases being eliminated is a bad thing?

If, say, we could eliminate every future occurrence of down syndrome, how is that bad?  Sure it would be bad if we used that capability to argue that we should eliminate living people with DS as well...but no one (sane) is arguing for that.

Realistically, the problem may be simple ignorance of the long-term effects of genetic engineering. Eliminating genetic diseases is an unambiguous good - but the repairs may, in turn, have unanticipated long-term effects that are not so good.

Same as any treatment, really. The issue will be the proper weighing of risks and benefits. The difficulty is that no-one can be sure about the risks until it is actually done, on humans.

[Barrister]

Assuming the research/application progresses in a regulated, transparent environment, what really is the downside to genetic engineering?  Humans will become "too good"?  Genetic diseases being eliminated is a bad thing?

If, say, we could eliminate every future occurrence of down syndrome, how is that bad?  Sure it would be bad if we used that capability to argue that we should eliminate living people with DS as well...but no one (sane) is arguing for that.

Because it's difficult to determine what is a "disease" that should be fixed.

The classic case is sickle cell anemia.  It is caused by having both parents being carriers of a single recessive gene, and the child inheriting the gene from both parents.  The problem is that being a carrier of only the single gene actually has some health benefits - carriers have greater resistance to malaria.

Or take downs syndrome (which now that I have twin DS nephews I'm learning more about).  It's a genetic disease, but it's quite different from something like sickle cell.  Sickle cell is caused by a specific defective gene.  Downs syndrome however is caused by having a duplicate chromosone.  It's not an inheritable gene - rather it's an error in early cell division.  As such there's no way to cure downs syndrome, either by today's or any readily forseeable future technology.  Instead the only way to "prevent" downs syndrome is to abort any pregnancy that tests positive for downs.

And finally, we would probably agree that dwarfism is a genetic disorder.  But what about someone who has the genes to be really, really short?  Where do you draw the line?

[crazy canuck]

Where do you draw the line?

The line is drawn in all research by the process of ethics approval which is required before any research is started.

[Barrister]

Where do you draw the line?

The line is drawn in all research by the process of ethics approval which is required before any research is started.

Which to date has not approved any alteration to human genomes.

[crazy canuck]

Where do you draw the line?

The line is drawn in all research by the process of ethics approval which is required before any research is started.

Which to date has not approved any alteration to human genomes.

But may.  Like our scientific knowledge, ethics approval is not static.

That is the main point of the article - carefully moving toward approval of research where ethically justified and legal.

[Fate]

Assuming the research/application progresses in a regulated, transparent environment, what really is the downside to genetic engineering?  Humans will become "too good"?  Genetic diseases being eliminated is a bad thing?

If, say, we could eliminate every future occurrence of down syndrome, how is that bad?  Sure it would be bad if we used that capability to argue that we should eliminate living people with DS as well...but no one (sane) is arguing for that.

You can't eliminate future cases of Down syndrome with genetic engineering. You can abort a fetus with Down syndrome or prevent a fertilized egg with Down syndrome from being implanted via pre-implantation genetic screening with IVF. But once the egg is fertilized/implanted and you decide to bring it term, the Down syndrome would be irreverisble.

Because it's difficult to determine what is a "disease" that should be fixed.

The classic case is sickle cell anemia.  It is caused by having both parents being carriers of a single recessive gene, and the child inheriting the gene from both parents.  The problem is that being a carrier of only the single gene actually has some health benefits - carriers have greater resistance to malaria.

And finally, we would probably agree that dwarfism is a genetic disorder.  But what about someone who has the genes to be really, really short?  Where do you draw the line?

We have treatments for malaria and are going to eventually eliminate it from the world. Sickle cell anemia is a horrible disease and it would be an unambiguously good thing for the world if we could elminiate it with genetic engineering.

Genetic engineering at the level we're talking about can't make short people tall or ugly people beautiful. It's going to be useful in curing single gene mutation genetic disorders. Things like cystic fibrosis, sickle cell anemia, thalassemia, Tay-Sachs, hemochromatosis. Traits like height, skin color, etc. are controlled by multiple genes which will not be amenable to simple engineering like a single point mutation would be.

[crazy canuck]

Genetic engineering at the level we're talking about can't make short people tall or ugly people beautiful. It's going to be useful in curing single gene mutation genetic disorders. Things like cystic fibrosis, sickle cell anemia, thalassemia, Tay-Sachs, hemochromatosis. Traits like height, skin color, etc. are controlled by multiple genes which will not be amenable to simple engineering like a single point mutation would be.

Sounds like a pretty strong argument in favour of approval. 

[Fate]

Where do you draw the line?

The line is drawn in all research by the process of ethics approval which is required before any research is started.

Which to date has not approved any alteration to human genomes.

There are two big categories of genetic engineering - germ cell line (heritable) and somatic (which are the non-reproductive cells and not heritable to the next generation.) Somatic genetic engineering has been approved for quite a while and is an active area of therapy in certain genetic disorders. Germ cell line genetic editting is where there's still an ethical question.

[Hamilcar]

And finally, we would probably agree that dwarfism is a genetic disorder.  But what about someone who has the genes to be really, really short?  Where do you draw the line?

Why shouldn't people be allowed to edit their own genes?

[DGuller]

And finally, we would probably agree that dwarfism is a genetic disorder.  But what about someone who has the genes to be really, really short?  Where do you draw the line?

Why shouldn't people be allowed to edit their own genes?

Same reason athletes shouldn't be allowed to dope.