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Sickle cell anaemia is the result of a point mutation. You have two routes to treat the patients. One is mRNA-based therapy (delivery of mRNA
Sickle cell anaemia is the result of a point mutation. You have two routes to treat the patients. One is mRNA-based therapy (delivery of mRNA without the point mutation). The other is gene editing on DNA level using CRISP-Cas9. Which one would you like to work on? Why do you choose it? Why do you abandon the other one? How can you achieve efficient delivery of the therapeutic nucleic acids you selected?
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