[candiodari]

Isn't that dangerous ? This will allow him to have a normal life, which includes kids, which will need this (potentially very expensive) treatment as well.

Wouldn't you, in order to not destroy the gene pool, want to restrict gene changes to both of those ? Either that or sterilize the patient to prevent transmission of lethal genes ? This treatment is VERY unfair to any children this guy fathers.

Of course I do get that the method used here goes not really support germ transfer (that requires the gene change to happen in vitro)

[vidarh]

Just carrying the sickle-cell trait is mostly neutral to highly advantageous depending on where you live, because it confers substantially increased resistance to Malaria.

This is how a trait that is so dangerous when expressed has managed to spread; in regions with endemic malaria, carriers have better survival chances and the difference is sufficient to make up for those who express it and end up dying young.

It is recessive - you need to inherit it from both parents to express it. So it is in fact entirely safe for this man to have children provided he has children with someone who is not a carrier, or ensure appropriate tests are taken during pregnancy.

Most people who carry this trait in developed countries today know about it, because the mechanism is very clear and they will usually know whether or not any of their family expressed the trait, and will have been tested to see if they inherited it. E.g. we had our son tested because his mother is a carrier, but because I am not we knew he could not have sickle cell disease.

My son is a carrier, but all that means is that like the guy in this article, he will want to make sure his partner is tested before having children, and to ensure to decide in advance what to do if both are carriers or if his partner has the disease (such as whether to have an abortion if tests show the child has the disease).

We can expect the number of people with this gene to drop over time because it is now so easy to avoid. But in the meantime a treatment would make a massive difference for those with the disease.

[PhantomGremlin]

This treatment is VERY unfair to any children this guy fathers.

In order to have the disease you need to inherit bad genes from both parents.[1] Consequently there are many more carriers than people who actually have the trait. So any of this guy's children have at most a 50% chance of having the trait if the other parent of the child doesn't have it, and 0% chance if the other parent isn't a carrier.

I'm sure if we sequenced your DNA we would find a number of potentially very problematic diseases for which you are a carrier. That applies to everyone, nobody has perfect DNA.

in order to not destroy the gene pool

There are a lot of morons in this world; many more being born every day. Many of them manage to reproduce before reaching the point where they would be under consideration for a Darwin Award. In the greater scheme of things this one particular genetic defect isn't seriously threatening to "destroy the gene pool".

[1] https://en.wikipedia.org/wiki/Sickle-cell_disease

[JshWright]

This is just a better treatment for sickle cell. Should sterilization be a requirement for any sickle cell treatment?

[jfarlow]

Part of the beauty of these kinds of gene therapies is that the cost can ultimately be lowered to near-zero levels. These are not drugs that must be taken over a lifetime, but can potentially provide 1-time delivery of the instructions to your own body about how to cure itself. Certainly in the near term the R&D must be paid for (and the marketing, and regulatory burden, and everything else). But in the long term the actual material used to change someone's life forever can cost pennies and a one-time delivery.