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by iamleppert 2777 days ago
You’re welcome and yes I agree about this being most impactful for children. Hemophilia had a big effect on my life, both physically and psychologically, as well as my family. It’s hard to grow up knowing you’re so different from others. Now, someone born with hemophilia may never even have to know. It changes things dramatically.

In a single generation we’ve gone from being able to treat the condition at all (replacement factor was just becoming available when I was born) to being able to completely cure it.

1 comments

a next step would be for the virus to fix the cells from which the gametes derive, then your future offspring would be cured as well