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A single injection that can alter the course of a genetic disease

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Collectively rare diseases are very common when you add them all up because there are many thousands of them and 80% of those rare diseases have a genetic basis. 

A solution works in a similar way to the Covid vaccine made by AstraZeneca. It uses a harmless virus to take a genetic message into the body. 

And that vector system is used to then ferry that genetic payload intravenously to the liver where it takes up residence, and hopefully, after a single injection, corrects that person's genetic abnormality for the rest of their life. 

https://www.abc.net.au/radionational/programs/healthreport/gene-therapy-rare-diseases/101405506



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