An experimental form of genome editing called clustered regularly interspaced short palindromic repeats or CRISPR/Cas9 could offer a faster and cheaper method of treating people with hemophilia, compared to the current DNA-editing method. Because hemophilia is due to DNA mutations, the genome editing could be used to efficiently slice at the DNA, removing or adding DNA to correct the mutation.
CRISPR/Cas9 has been trialed on mouse models who had their clotting factor IX removed. The mice showed normal levels of IX production for four months following treatment.
This research may lead to new techniques being developed for the treatment of humans with genetic diseases such as hemophilia. Find out more about this research here.
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