Human trials recently began to use the genome editing technology CRISPR to treat sickle cell anemia using edited stem cells. Sickle cell anemia is caused by a single DNA Mutation, and is also a disorder of the red blood cell. Given that this is a more simplistic genetic issue to address (i.e. there is only one DNA mutation to correct), and that the disorder affects an easily replaceable connective tissue, is the application of CRISPR limited as a treatment? Could CRISPR be used to treat disorders that are a results of multiple DNA mutation, and that affect a different types of tissue?

  • $\begingroup$ Welcome to Biology.SE, if you haven't done so already you can take the Tour to learn a bit more about the site. Also please not that your statement "The disorder affects an easily replaceable connective tissue" is wrong. The textbook version of sickle cell anemia affects red blood cells, which are neither connective tissue nor easily replaceable (as a whole system). $\endgroup$
    – Nicolai
    Nov 14 '18 at 9:15

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