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So CRISPR is definitely a hot topic right now, and we hear that it has better success in vitro rather than in vivo (correct me if I'm wrong), but how far out is

So CRISPR is definitely a hot topic right now, and we hear that it has better success in vitro rather than in vivo (correct me if I'm wrong), but how far out is this to being available as a therapeutic mechanism in the treatment of diseases? And what diseases are those?

entee 10y ago
A major issue is that in vitro you have the luxury of many attempts to get the right result. Here's a somewhat simplified but illustrative example: Say you have 10 mouse embryos. You use CRISPR, then check them for whether it worked out as expected. Only one of those embryos is as desired. That's totally fine, and massively accelerates lab research. But you can't apply those numbers to something like human embryos ethically. I think (and people I know in academic science agree) that CRISPR is awesome for research science, but that it's going to take some time before it's directly useful in the clinic. And even when it is, it's likely to be useful in a subset of cases where you can check whether CRISPR had the desired effect before introducing the results into a patient. This is still very useful (immunotherapy for cancer for example, another very hot topic these days), but doesn't quite match the breathless statements from the mainstream press. This work is really nice because it likely reduces the number of embryos (in the above contrived example) you'd need to screen before finding an appropriate one. On the other hand it's still very very hard to get this to work when you have a limited number of cells you can apply CRISPR/CAS to. Progress, yes. Panacea, no.
- codeulike 10y ago
  And I would have thought that letting an agent that can modify DNA loose anywhere except strict lab conditions could be dangerous? Do you expect gov regulations to happen soon in this area?
  - adrianN 10y ago
    There are so many agents that can modify DNA around you that one more probably doesn't make a difference.
  - tstactplsignore 10y ago
    Not dangerous at all. Requires high concentrations of reagents to function, can't penetrate cells on it's own, sensitive to pH and solution conditions, protein doesn't aerosolize on it's own, etc. The government regulates how you can use it, but cas9 itself is harmless

entee 10y ago

A major issue is that in vitro you have the luxury of many attempts to get the right result. Here's a somewhat simplified but illustrative example: Say you have 10 mouse embryos. You use CRISPR, then check them for whether it worked out as expected. Only one of those embryos is as desired. That's totally fine, and massively accelerates lab research. But you can't apply those numbers to something like human embryos ethically. I think (and people I know in academic science agree) that CRISPR is awesome for research science, but that it's going to take some time before it's directly useful in the clinic. And even when it is, it's likely to be useful in a subset of cases where you can check whether CRISPR had the desired effect before introducing the results into a patient. This is still very useful (immunotherapy for cancer for example, another very hot topic these days), but doesn't quite match the breathless statements from the mainstream press. This work is really nice because it likely reduces the number of embryos (in the above contrived example) you'd need to screen before finding an appropriate one. On the other hand it's still very very hard to get this to work when you have a limited number of cells you can apply CRISPR/CAS to. Progress, yes. Panacea, no.

codeulike 10y ago
And I would have thought that letting an agent that can modify DNA loose anywhere except strict lab conditions could be dangerous? Do you expect gov regulations to happen soon in this area?
- adrianN 10y ago
  There are so many agents that can modify DNA around you that one more probably doesn't make a difference.
- tstactplsignore 10y ago
  Not dangerous at all. Requires high concentrations of reagents to function, can't penetrate cells on it's own, sensitive to pH and solution conditions, protein doesn't aerosolize on it's own, etc. The government regulates how you can use it, but cas9 itself is harmless