We have seen only recently how devastating a diagnosis like this can be, with the heartbreaking story of Charlie Gard generating headlines worldwide.
The 11-month-old died last week from a genetic condition called mitochondrial DNA depletion syndrome 13, which triggers progressive muscle weakness and eventual brain damage.
It is cases like Charlie’s motivating scientists all over the world to further understand our genetic make-up in the hope we could one day eradicate, or at least treat, all diseases relating to our genes.
That is why yesterday’s breakthrough news is a huge step in the right direction.
Researchers from Oregon Health & Science University in the US successfully snipped away faulty DNA in 42 out of 58 embryos which had a predisposition to a potentially fatal heart condition.
They used gene editing technique CRISPR which, when injected into an egg along with a sperm, acts like DNA spell-check software to find a specific faulty gene and replace it with a healthy version.
This would theoretically allow a baby to be born without hypertrophic cardiomyopathy, the biggest cause of sudden death.
The technique’s recent success gives hope to scientists — and to families who have experienced heartbreak through the generations after losing relatives to genetic diseases.
But we need to be absolutely sure it is safe and the benefits outweigh the risks.
But this couldn’t be further from the truth. There are strict regulations which ensure experiments like this are carried out in highly controlled environments.
The Human Fertilisation & Embryology Authority (HFEA) has tight testing requirements that ensure it will only be rolled out if proven medically worthwhile.
And laws have to be passed before it could be offered at all in the UK and on the NHS.
Besides, we still know very little about what our genes do and which genes are linked to which physical and mental traits.
There are hundreds of genes linked to height and thousands linked to intelligence.
We would not know where to begin when tasked with producing a “designer” baby.
And we have little motivation to do so anyway.
All scientists understand the ethical considerations that surround tampering with genes — and we focus on understanding our genome to help eradicate disease, not for vanity purposes.
More research needs to be done before we will see babies born free of genetic diseases to families with a history of it.
If proven successful, I hope it will happen in my lifetime.
What we won’t see is researchers snipping up bits of DNA to make sure babies are born with a specific hair colour, IQ or sporting ability.
It is a risky procedure and should only be carried out if there is medical need.
We certainly won’t ever fiddle around with an embryo’s genes as standard.
The role of scientists is to find out how to do things to better our health.
It is up to regulatory bodies and the public to determine how, or if, we should use it.
Advances in science like this are driven by our wish to reduce the burden of disease and to make us healthier.
After all, knowledge is power. But it is not the scientists alone who wield that power, it is society as a whole.
In future, thanks to science, diseases that have blighted families for generations could be wiped out.
But we are a long way from establishing whether gene editing like this is safe, and the whole scientific community takes testing of new techniques like this exceptionally seriously.
The US trial has raised important questions about CRISPR and we are a long way from properly understanding it.
For example, we don’t know how the trial embryos managed to take copies of relevant healthy genes from the egg after the faulty one was snipped away.
Neither do we know the implications on future generations of editing genes in this way, so much more research needs to be done before families could benefit.
It could take years to establish whether this is safe.
Besides, it is still illegal to transplant embryos with edited genes into a womb.
There are currently some techniques available to people who know genetic diseases run in their family.
Preimplantation genetic diagnosis (PGD) screens embryos fertilised in the IVF process for genetic disease. Only healthy ones can be implanted.
But it is not completely foolproof and cannot be used in every case, so we need to develop new techniques, like CRISPR. Our intentions are to do good, not create the “perfect” race.
Those working in this field are doing so because they are concerned about the suffering caused by genetic disease.
No parent should have to lose a child to something that science could help prevent.
I hope this research can be developed responsibly so no parent has to.
Source: http://bit.ly/2u6qcNf
