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gthing t1_iy1ljxx wrote

Reply to comment by FogeltheVogel in How exactly does CRISPR-CAS9 insert new genes? by AutomaticAd1918

If my 23 and me shows me I have certain genes that are associated with higher risk for X,Y,Z - are those theoretically then curable with CAS9? Are the genes even understood enough to say if we switch one off it's not going to have some cascading weird effect or even that it will actually cure you? Last question: how long in your wild estimate until most everyday gene disorders are routinely cured during childhood?

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wilnyb t1_iy27zw0 wrote

A major issue with correcting genes in adults is the the large number of cells that needs to be corrected. Let's say you have a muscle disease, hitting every single muscle cell in you body to correct an genetic disorder is very complicated. As of right now, this is easier with blood cells. You can isolate hematopoietic stem cells, that you can edit and then reintroduce in a patient. Those cell will repopulate the immune system.

Every disease is different. For many diseases it might be enough to correct 10% of the cells for a patient to be able to live with the disorder. Some of those examples already exist today. Some more complicated genetic disorders we might never be able to correct (at least in our life time).

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HotDadBod1255 t1_iy36nd2 wrote

Unfortunately there really isn't much clinical data for in vivo gene editing, the only results so far are from two clinical trials from Intellia Therapeutics. Their results are really promising, but so far they've only shown they can do gene knockout in liver cells, which is pretty limited in scope. Hopefully them and some others are working on other ways to perform gene editing.

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