https://www.earth.com/news/crispr-used-to-remove-extra-chromosomes-in-down-syndrome-and-restore-cell-function/

Thanks for posting, that is an interesting article.  I wonder if we will ever be at the point where a CRISPR like technology could be used to alter the chromosomes of a living creature or even an early fetas with a genetic disorder?  The idea opens up a lot of philosophical questions.  What happens to an individual who goes the genetic alterations?  Is that the same person after that, or is the different DNA creating a new (replacement) person?  It is an interesting thought, even if the altered person had all the same memories or might be perceived as remembering someone else's life.  I also wonder if personalities will change along with the physical differences?  Are our "souls" in our DNA?

May 2025:

"For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy scientists tailored to specifically repair his unique mutation."

A bespoke gene-editing treatment for a baby born with a life-threatening disorder : Health News : NPR
https://www.npr.org/sections/shots-health-news/2025/05/15/nx-s1-5389620/gene-editing-treatment-crispr-inherited

They were able to use CRISPR to edit the genes specifically in the liver in that example -- a localized change, which would be very different than modifying the entire body's DNA to reverse Down Syndrome.   RC, you probably know way more about this topic than me.  In the example of the baby getting his liver modified, with that child's body now treat his liver as a foreign object like it would with a liver transplant?  Will the boy have to be on immune suppressors for the rest of his life?

Good question.

I don't know, but Debbie thinks that long term immune suppressors will not be required.  Though modified, the cells involved are still his own.  This guess is based on the fact that some transplant clinics are dropping immune suppressors if the donor is close family. There's one guy in /r/transplant who was never put on them and that was 10-15 yrs ago --UCSF Transplant Center, IIRC.

>philosophical questions

I don't have answers to your questions, littleman. But I doubt many parents will say "no thanks" to the cure.

>But I doubt many parents will say "no thanks" to the cure.

Just to clarify, those questions wouldn't stop me from using gene editing tech to save a life of a loved one.  I am only thinking about those questions because I am not in that situation myself.

I had a cousin with Down's syndrome. His symptoms were severe, and my aunt and uncle were told he would likely live less than two years. They were advised to put him in an institution and forget him.

They said no to that, he's ours and we will love him for whatever time he has.

Dougie was intellectually challenged, and the challenge was made harder because his speech was hard to understand. His tongue was too big and there were some sounds he simply could not say.

He went to a special school and among other things learned to print. He would spend hours in his room printing the names of NHL players. He was proud to show you his lists!

He never reached normal adult height, and one of the most poignant moments in the story was when my aunt realized that he was asking why his cousins were all growing up and getting big, but he wasn't.

He loved music, and someone showed him how to play the spoons. What he lacked in finesse, he made up in joy. He also learned to dance. With help from a family member, he would attend dances at one of Winnipeg's institutions for the developmentally disabled. He was a popular dance partner, and had a gift for bringing shy people out of their shells.

No matter who you were, Dougie would greet you with a cheerful "Hi!" (a word he could articulate) and a handshake. He was neither impressed nor unimpressed by worldly accomplishments, he just liked who you were.

In maybe the last decade of his life, he learned some ASL sign language. It made communication easier, and we all wished that he ... and the family ... had had some signing instruction years earlier.

The infant with the two-year prognosis made it to age 53. It was a testament to the love and care of his family.

His funeral was packed. Near the end of the service, the celebrant asked if anyone would like to say a few words. I've been at funerals where maybe one or two people had something to say at that juncture, or maybe no one.

This time, a woman stood, remarked on Dougie's loving heart, and said, "I need to be more like Dougie." Someone else stood and said something similar. And someone else, and someone else ... I lost count after two dozen people. A man dressed in what I'm sure was a thousand-dollar suit stood and said, "I need to be more like Dougie. I need to learn from Dougie." The next funeral which was scheduled in the chapel did not start on time.

As I type this I'm reminded that I too need to be more like Dougie. A little man with a big heart.

Dougie had Down's syndrome, but he was as beautiful as any human who ever lived.

Would CRISPR treatment have been a good thing? Hard to say ...

>> same person after that, or is the different DNA

There might be a clue in the situation of conjoined twins. They share one body and the same DNA, but they're two separate people.

DNA is certainly part of one's personhood but it's not the whole story.

>But I doubt many parents will say "no thanks" to the cure.'

I think the philosophical questions come in when you start to broaden the definition of disease. Is Down's Syndrome a disease? Is being slightly below average intelligence a disease?  Is being shorter than average? Are small boobs a disease?

I'm being stupidly provocative, but when you can order a customized child, it raises big philosophical questions about where to draw the line.

I once started (alas never finished) a short story that was a first-person narrative of a "natural" in a world where everyone's parents had opted for the genetic therapy that made their kids not need to sleep. Very important if you want your kid to get ahead in life. Totally irresponsible for any parent to let their kid grow up needing a natural amount of sleep when there's a solution for that.

That said... it seems like celiac could be a candidate and that would be incredible. Maybe Alzheimer's. I can't see the world being impoverished by being rid of those diseases.

But again, where you draw the line and what qualifies as a disease raises some huge ethical issues.

In some ways, vaccines are a good model - babies don't get to decide whether or not to be vaccinated and in general, most of us are in favor of saving them from those diseases that can be prevented with vaccination. But even there, opinions differ... even at the, ahem, highest levels of government.

>> alas never finished

So dust it off and finish it now.

>> So dust it off and finish it now.

It didn't have legs. It was an idea, but I didn't know where it would go. In general, I don't think long-form fiction is my thing.

Stephen King once said once he had made it as an author, people would come up and say, "I have a great idea for a book. I'll tell you what it is and all you have to do is write it and we'll split it 50/50."

His response? "I have 10 ideas for books every single day. The hard part is the writing it."

>> didn't have legs

Then don't say "alas" when telling us that it didn't get finished! 

My favourite CEO said to me once that good ideas are easy, but it's only good execution that puts money in your pocket.

As the gambler said: Know when to fold 'em