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Teenager's sickle cell reversed using a treatment to change his DNA

bbc.com

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Re: Teenager's sickle cell reversed using a treatment to change his DNA

#3
post #2

Will this change potentially be transmitted to his children?

It's unlikely. The virus used to modify the bone marrow cells would have to stay active after the transplant and infect the rest of his body (specifically his testicles).

Re: Teenager's sickle cell reversed using a treatment to change his DNA

#5
post #2

Will this change potentially be transmitted to his children?

It's unlikely. The virus used to modify the bone marrow cells would have to stay active after the transplant and infect the rest of his body (specifically his testicles).

It sounds like this research involves somatic gene transfer, in which the change to the genome is not passed on to children:

Gene transfer can be targeted to somatic (body) or germ (egg and sperm) cells. In somatic gene transfer the recipient's genome is changed, but the change is not passed on to the next generation. In germline gene transfer, the parents' egg and sperm cells are changed with the goal of passing on the changes to their offspring.

https://www.genome.gov/10004764/germline-gene-transfer/

Re: Teenager's sickle cell reversed using a treatment to change his DNA

#7
post #2

Will this change potentially be transmitted to his children?

Unfortunately, human germline modification is still taboo here: https://motherboard.vice.com/en_us/article/scientists-argue-...

It angers me that a misguided and stringent sense of ethics might slow the deployment of one of the most beneficial technologies ever conceived.

Re: Teenager's sickle cell reversed using a treatment to change his DNA

#8
post #2

Will this change potentially be transmitted to his children?

Unfortunately, human germline modification is still taboo here: https://motherboard.vice.com/en_us/article/scientists-argue-... It angers me that a misguided and stringent sense of ethics might slow the deployment of one of the most beneficial technologies ever conceived.

I agree with this, but not a popular opinion. Hence the down votes you are getting. Korea and China don't have the same memetic immune response to such ideas, injecting one's children with growth hormone in hope of increasing future job prospects, for example, is extremely common in Korea. I suspect embryo selection and generic engineering for increased IQ will take off in East Asia first. Then we will be forced to allow it to compete. 50+ more IQ points looks very possible: https://arxiv.org/abs/1408.3421

However, any significant lag could spell the end of American economic dominance. For this reason, I intend to start investing more in Korean firms in the next few decades.

Re: Teenager's sickle cell reversed using a treatment to change his DNA

#9

here is the nejm paper in the article: http://www.nejm.org/doi/10.1056/NEJMoa1609677

Ultimately, they are delivering a payload with a slightly modified version of wild-type hemoglobin (a single point mutation from wild-type). Patients with sickle-cell disease have a (different) mutation in their hemoglobin that causes the hemoglobin protein to aggregate, and not carry oxygen. The modified version being delivered to patients is not just a 'corrected, wild-type' version of the protein capable of carrying oxygen more efficiently, but actually carries a synthetic mutation that actually prevents the patient's version from aggregating. So if even 20% of the hemoglobin in the treated patient's blood is of this modified type, then none of their hemoglobin will aggregate, and they should get much of their oxygen-carrying capacity back. This modification to the introduced version of the protein also allows it to be easily identified and tracked to monitor things like its concentration.

The actual sequence and mutation HBB [T87Q] protein: https://serotiny.bio/pinecone/part/10803

A short little writeup I did about the mutation: https://serotiny.bio/notes/proteins/hbb/

A very detailed presentation about Bluebird Bio's therapy (called LentiGlobin BB305) provided to the government: (PDF warning) http://osp.od.nih.gov/sites/default/files/1164_bluebirdbio.p...

Re: Teenager's sickle cell reversed using a treatment to change his DNA

#10

Earlier quoted context omitted.

Unfortunately, human germline modification is still taboo here: https://motherboard.vice.com/en_us/article/scientists-argue-... It angers me that a misguided and stringent sense of ethics might slow the deployment of one of the most beneficial technologies ever conceived.

I agree with this, but not a popular opinion. Hence the down votes you are getting. Korea and China don't have the same memetic immune response to such ideas, injecting one's children with growth hormone in hope of increasing future job prospects, for example, is extremely common in Korea. I suspect embryo selection and generic engineering for increased IQ will take off in East Asia first. Then we will be forced to a…

A better way to increase iq would be to raise the socioeconomic level...
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