What is gene editing (CRISPR/Cas9)?
CRISPR/Cas9 is a versatile tool for correcting mutations in DNA. There are several different ways to use it. Two examples are shown below:
Correction of a Deletion Mutation using CRISPR/Cas9
Here is an example of an individual with Duchenne missing exon’s 6 and 7, showing how this technique corrects this deletion by inserting the missing exons.
This approach could work for any deletion. It’s just a matter of changing the payload, so it is specific for the mutation you are wishing to correct.
In this second example we are looking at an individual with Duchenne because of an exon 6 duplication.
This technique finds this duplication and essentially cuts it out, so the gene can once again function and produce fully functional dystrophin.
As you can see from these examples, CRISPR/Cas9 would be a very individualized treatment or would be specific for very small groups of patients. Right now, there are no clinical trials in this category, but it is an exciting area of preliminary research.