[dclowd9901]

As the brother of a young, amazing man who we lost due to another genetic ailment (CF), whenever I see stories like this, it makes me so hopeful for families in the future never having to see a loved one deteriorate due to a bad roll of the dice.

Hats off to everyone out there putting in the hours to make the lived experience of these folks much better than they would have otherwise been. If only we had more of you in the world.

[unglaublich]

Completely agree, this is why we need to detect bad rolls and give people the ability to reroll.

[ASalazarMX]

Since this is genetic theraphy, it would be amazing if it corrected the bad roll permanently. Being in-utero, this might be the case here.

[armadsen]

Spina Bifida is not primarily a genetic disease. It's caused by a failure of the neural tube in the developing embryo to close fully. No one knows the exact causes, but folic acid deficiency in the mother before and during pregnancy makes it more likely. It also seems to run in families a little, but only weakly, and we haven't identified any specific genetic cause. This treatment is very promising, but it's not a cure, just a (hopefully) even better treatment than the existing in-utero surgery that doesn't include stem cells.

[tclancy]

Can you expand on what you mean by that? Otherwise there’s a way to interpret it that is less than lovely.