CRISPR Medical Applications

It looks very promising. The trick is making sure it does what it is supposed to do and doesn't do something it isn't supposed to.

It has to be considered "safe", but someone with a terminal disease may have a different definition of "safety".

It's not a magic bullet, for sure. There was improvement, if not a cure. For a terminal or disabling disease, those might be the kind of results that would tempt you to take the risk. More likely it will be refined in future and become a therapy of choice.

That is unless there turn out to be some unforeseen and serious adverse side effects. I guess we don't even know how well these reconstructed genes will hold up over time, either.

I thought the images of normal structure vs the restoration of gene function with crispr was interesting. Obviously not a one-one match. I wonder why.

This is but one important step in showing how helpful gene therapy can ultimately become.

IMHO I suspect gene therapy will ultimately be most useful for young patients, possibly in-vitro. Correcting errors in a "blue print" pattern will work best when the body has yet to learn how to compensate for a flaw/feature.

The paradox of the resistance to cochlear implants in some deaf families may come to play, too.

{That's not well explained but I have to go.}

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