Could Genome Editing Be a Future Treatment for Hemophilia?

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.

MORE: Bioverativ’s therapy donations are part of the buzz for World Hemophilia Day.

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.

MORE: Phase 3 trials evaluating further potential of two treatments for hemophilia A.

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