3 ms·
So it is today technically possible to run a gene therapy in adult humans via CRISPR and some recent innovations in vectors with a good expectation that tissue
by reasonattlm 11y ago
So it is today technically possible to run a gene therapy in adult humans via CRISPR and some recent innovations in vectors with a good expectation that tissue coverage is going to be what you want it to be - i.e. enough cells take up changes for it to work. (See for example http://today.duke.edu/2015/12/crisprmousedmd http://today.duke.edu/2015/12/crisprmousedmd for the good tissue coverage angle).
This is going to be next year's medical tourism, analogous to the progression of stem cell therapies from the turn of the century.
There are a few really obvious candidates, such as myostatin and follistatin to greatly increase muscle growth - and at least one person who has had that done already, c.f. BioViva. To do this all you need are the connections. Buy into a biotech startup company, make the arrangement with a lab in Mexico or Thailand and off you go. If you knew the right people, you could be on a plane tomorrow and the beneficiary of elective gene therapy the day afterwards.
It can't be overstated how easy CRISPR makes this. It is easier even than induced pluripotency, and that spread like wildfire through the labs when it emerged.
The more interesting thing to me is that there are probably a hundred less obvious, poorly studied, poorly followed up, but very interesting genetic alterations that could be done, and probably will be done largely outside the institutions of medical research. This is what happens when cost falls. Any single gene is fair game now. Want great resistance to ischemia/reperfusion injuries? Knock out PHD1 [1]. Want your aged liver to function as well as it did when you were young? Add more lysosomal receptors. [2] Want to have permanent operation of the benefits of fasting and exercise in the form of upregulated autophagy? Increase AMPK levels [3]. And so on and so forth through scores of genes. Maybe many of them will work as the studies suggest, maybe not.
[1]: http://www.vib.be/en/news/Pages/-VIB-researchers-discover-possible-strategy-against-stroke.aspx http://www.vib.be/en/news/Pages/-VIB-researchers-discover-po...
[2]: http://www.nytimes.com/2009/10/06/science/06cell.html http://www.nytimes.com/2009/10/06/science/06cell.html
[3]: http://www.salk.edu/news-release/how-the-cells-power-station-survives-attacks/ http://www.salk.edu/news-release/how-the-cells-power-station...