Gene therapy - a brave new world?

[Rum]

From: http://www.bbc.co.uk/news/health-40802147

Scientists have, for the first time, successfully freed embryos of a piece of faulty DNA that causes deadly heart disease to run in families.

It potentially opens the door to preventing 10,000 disorders that are passed down the generations.

The US and South Korean team allowed the embryos to develop for five days before stopping the experiment.

The study hints at the future of medicine, but also provokes deep questions about what is morally right.

Science is going through a golden age in editing DNA thanks to a new technology called Crispr, named breakthrough of the year in just 2015.
Its applications in medicine are vast and include the idea of wiping out genetic faults that cause diseases from cystic fibrosis to breast cancer.

Heart stopper

US teams at Oregon Health and Science University and the Salk Institute along with the Institute for Basic Science in South Korea focused on hypertrophic cardiomyopathy.

The disorder is common, affecting one in every 500 people, and can lead to the heart suddenly stopping beating.

It is caused by an error in a single gene (an instruction in the DNA), and anyone carrying it has a 50-50 chance of passing it on to their children.

In the study, described in the journal Nature, the genetic repair happened during conception.

Sperm from a man with hypertrophic cardiomyopathy was injected into healthy donated eggs alongside Crispr technology to correct the defect.

It did not work all the time, but 72% of embryos were free from disease-causing mutations.

Eternal benefit

Dr Shoukhrat Mitalipov, a key figure in the research team, said: "Every generation on would carry this repair because we've removed the disease-causing gene variant from that family's lineage.

"By using this technique, it's possible to reduce the burden of this heritable disease on the family and eventually the human population."

There have been multiple attempts before, including, in 2015, teams in China using Crispr-technology to correct defects that lead to blood disorders.

But they could not correct every cell, so the embryo was a "mosaic" of healthy and diseased cells.

Their approach also led to other parts of the genetic code becoming mutated.

Those technical obstacles have been overcome in the latest research.

However, this is not about to become routine practice.

The biggest question is one of safety, and that can be answered only by far more extensive research.

There are also questions about when it would be worth doing - embryos can already be screened for disease through pre-implantation genetic diagnosis.

However, there are about 10,000 genetic disorders that are caused by a single mutation and could, in theory, be repaired with the same technology.

Prof Robin Lovell-Badge, from the Francis Crick Institute, told the BBC: "A method of being able to avoid having affected children passing on the affected gene could be really very important for those families.

"In terms of when, definitely not yet. It's going to be quite a while before we know that it's going to be safe."

Ethical?

Darren Griffin, a professor of genetics at the University of Kent, said: "Perhaps the biggest question, and probably the one that will be debated the most, is whether we should be physically altering the genes of an IVF embryo at all.

"This is not a straightforward question... equally, the debate on how morally acceptable it is not to act when we have the technology to prevent these life-threatening diseases must also come into play."
The study has already been condemned by Dr David King, from the campaign group Human Genetics Alert, which described the research as "irresponsible" and a "race for first genetically modified baby".
Dr Yalda Jamshidi, a reader in genomic medicine at St George's University of London, said: "The study is the first to show successful and efficient correction of a disease-causing mutation in early stage human embryos with gene editing.

"Whilst we are just beginning to understand the complexity of genetic disease, gene-editing will likely become acceptable when its potential benefits, both to individuals and to the broader society, exceeds its risks."

The method does not currently fuel concerns about the extreme end of "designer babies" engineered to have new advantageous traits.

The way Crispr is designed should lead to a new piece of engineered DNA being inserted into the genetic code.

However, in a complete surprise to the researchers, this did not happen.

Instead, Crispr damaged the mutated gene in the father's sperm, leading to a healthy version being copied over from the mother's egg.

This means the technology, for now, works only when there is a healthy version from one of the parents.

Prof Lovell-Badge added: "The possibility of producing designer babies, which is unjustified in any case, is now even further away."

[Brian Peacock]

Perfection here I come!

[JimC]

If it were limited to correcting single gene defects, which produce a clear genetic disease, replacing them with the normal variant, and if it eventually became both routine and free from any side effects, there would not, IMO, be any ethical issues.

[Strontium Dog]

I would go even further than that; so long as you're not making changes that will limit or disable future generations (eg just look at what we have done to the noble wolf, and all the health issues the various breeds have to endure) then I think it's fair game.

[JimC]

I would go even further than that; so long as you're not making changes that will limit or disable future generations (eg just look at what we have done to the noble wolf, and all the health issues the various breeds have to endure) then I think it's fair game.

Going beyond the straightforward corrections I mentioned earlier would have to be thought through very, very carefully, as there is a potential minefield of unintended consequences and ethical concerns. I'm not saying that genetic alterations beyond simple corrections of defects are automatically wrong, but I'm saying they could easily be seriously problematic.

[Brian Peacock]

if only they could make me purr like a cat and photosynthesise.

[JimC]

Greenie Pussy!

[pErvinalia]

Just saw this on the news. Rolled my eyes at the mention of ethical questions. Fuck off wankers.

[JimC]

You don't think that there is a chance that unrestricted use of genetic alteration of human embryos could run into some problematic areas? I know that the god-botherers often use the smokescreen of "ethical issues" to hide their fundamentalist views on the sanctity of life and the rest of the usual nonsense, but that does not mean that there are no ethical issues for rational consideration, if the genetic alterations go beyond repairing clear faults.

[pErvinalia]

yeah, but the wanker in the article was complaining that fixing this defect was an ethical issue. It's no more of an ethical issue than cutting out cancer from a live person. As you say, there's going to be a fair bit of religious nonsense infect this issue.

[JimC]

yeah, but the wanker in the article was complaining that fixing this defect was an ethical issue. It's no more of an ethical issue than cutting out cancer from a live person. As you say, there's going to be a fair bit of religious nonsense infect this issue.

Fine; I thought you were meaning ethical issues about the whole area, not just the straightforward repair, where I agree it should be non-problematic, as long as there are no biological side effects...

[pErvinalia]

Yeah, I guess the contentious point is how exactly a "defect" is defined. Also unintended side effects. I don't really know enough about it to know how possible weird shit down the line is after something like this.

[Tyrannical]

Too lazy to find the episode of The Outer Limits, but it doesn't end well.

[Rum]

I also wonder if it might just be available to those who can afford it - assuming it isn't affordable to national health systems. That raises the prospect of an elite rich and healthy class and all that goes with it..this the 'Brave new world' in the OP title.

[Animavore]

I bet you it'll be the Christian right as usual railing against it, despite the fact it will reduce abortions. I think something like 90% of children born with Downs Syndrome are aborted.