0
$\begingroup$

Edward Lanphier, CEO of Sangamo BioSciences mentions here(https://youtu.be/dJ1B8XnyEnM?t=1215) that scientists can now specifically target cells, change the genome within a cell. Say you want to cure a type of disease, which can be linked to your genome. You have successfully removed the faulty gene in your genome (GenomeA), replaced it with a correct gene resulting in a improved genome (GenomeB) in a single cell. The next step will probably be to put it back into the human body. However, as I understand it every human cell contains your faulty human genome (GenomeA).

Question:

  1. Even if you put a few cells with the correct genome sequence (GenomeB) into your human body, it is probably going to be overwhelmed by the other cells that contain your faulty genome (GenomeA). Cells containing the correct genome (GenomeB) will divide and multiply but so will cells containing the faulty genome (GenomeA). In this “genomic war” will the fewer cells containing the correct Genome (genomeB) ever win? Or will you need period infusions of cells with the correct genome "forever"?

How would you guys think about this?

Optional questions:

  1. Even if it wins, it will probably take a very long time to completely replace all the cells with the faulty genomes. How much time is required before you can completely replace all the cells with the incorrect genomeA, with cells containing the correct genomeB?

  2. Say you realize that you have a faulty genome (GenomeA), and you attempt to replace your genome with a correct genome (GenomeB). After 2 years, you have another disease and realize that you have to again correct your genome with GenomeC. However, 2 years is not enough to replace all your cells with the incorrect GenomeA, with the correct GenomeB. If you attempt to undergo genetherapy, now you will have to introduce another group of cells with GenomeC. Now you have cells with multiple different types of genomes floating around: genomeA, genomeB and genomeC. Are there any complications that happen due to the presence of multiple types of cells with different genomes in your body?

$\endgroup$
  • $\begingroup$ good questions... but way too many posed in a single entry... can you try and focus your question? it's very unlikely that anyone will answer all of them in one post. $\endgroup$ – Vance L Albaugh Feb 4 '17 at 19:13
  • $\begingroup$ I feel the question is useful and raises good points and those points shouldn't be removed. Even if a person may not answer all of them in one post, he or she may answer some of them, with other answers to be obtained in other posts. In terms of the question, I don't see why only a few cells can be put into the human body. What technology can do to one cell at a time now, it should be able to do to thousands or millions at a time in the future. $\endgroup$ – sterid Feb 5 '17 at 9:24
  • $\begingroup$ Hi Vance, you are right, my question is a bit broad, since my specialization is not biology/gene-therapy. Since 2 people pointed out that the question is a bit broad, I only highlighted the first question and left the 2nd and 3rd questions as optional. It kind of a compromise, so as to address Sterid's request as well. $\endgroup$ – alpha_989 Feb 6 '17 at 16:20
1
$\begingroup$

Your question is too broad. It is better to take some specific example. Not all cell's in the body divide. If you take cell out of the body and want to return it back, it must divide. So we probably could talk about stem cells for example. The stem cells don not really sit in one place. And it almost impossible now to get such cells from the place that they sit in it, and then return in back. It is possible with bone marrow for example.

If we take some example, like ALS - some percent of the disease is thought to be genetic. But the mutation is in all the body cells. Mostly it's affects astrocytes, microglia and motor neurons. You cant take them out of the body, fix and return back. The only chance to fix is to work inside the body. For example by using some sort of virus (AAV for ex.) And CRISPR.

However, if we follow your story and we insert cells with genomB, they will live together with cells with genomA, if there is not some bigger mortality of the original cells.

$\endgroup$
  • $\begingroup$ Thanks for pointing out some use cases. My specialization is not biology, and I am coming in from a 30000 feet so you are right that the question is perhaps a bit broad. I guess my intuition was kindof right. It seems you do need some kind of periodic infusions of modified cells or viruses carrying the modified genes, as pointed out by this article. nytimes.com/2005/07/03/books/chapters/more-than-human.html?_r=0 $\endgroup$ – alpha_989 Feb 6 '17 at 16:17

Your Answer

By clicking “Post Your Answer”, you agree to our terms of service, privacy policy and cookie policy

Not the answer you're looking for? Browse other questions tagged or ask your own question.