Holy Shit, Gene Therapy!

[Arathena]

So, there's a rare genetic disease passed down on the X chromosome from mothers, where it is recessive, to sons, where the Y chromosome cannot compensate for it, called SCID-X1, in which the body's natural state is equivalent to end-stage AIDS.

Well, they pulled some stem cells from 9 babies with SCID-X1 in 1999-2000, used a retrovirus to put in a gene that made the protein they were missing, and stuck the cells back into them. They canceled further experimentation almost immediately, because the retroviruses did what retroviruses do, and gave 4 of the kids leukemia. However, 10 years later, 8 of the kids are alive. All 8 have functioning immune systems.

Science win.

[Ddrak]

Nice, if somewhat scary in the possibilities.

Dd

[Arathena]

Every new technology has its moral issues, and it's risk vs reward calculations - as well as its subtle limitations.

For example - this therapy can effectively add a given protein or set of proteins to an individual, in a way that they are not likely to be able to pass it down in reproduction. It cannot cause a person to subtract a protein. (Though it can theoretically add an inhibitor, or a competing protein it gets very messy very quickly). Engineered transhumans aside, the more immediate and intriguing question is: What diseases are worth a 40-50% chance of cancer involved in the cure? SCID-X1 is a fairly open and shut case - People that have it die fast, and the ones that live have pretty miserable bubble-lives. But let's take another case.

Preliminary studies of the autism spectrum indicate that the major issue is the genetic/epigenetic derailment of a broad developmental system related to the socalization centers inside the brain. This can occur in hundreds, if not thousands of modes, but many of them may be correctable by this technique, if caught early enough. Now, the question is: Is bringing a person to social norms worth, say, a 50% chance of cancer?

[Taxious]

Is bringing a person to social norms worth, say, a 50% chance of cancer?

Interesting question there - I would say yes for me personally but it'd be hard to make that decision for someone else.

[Minute]

I'm all for stem cell research, I think it's great, but being a total product of our movie industry anything with the word retrovirus in it means that soon some 28 days later shit is going to happen.