We finally have some very promising news for the development of adequate treatment for our boys with mutations requiring double or multi exon skipping!

In reading through this article about injecting a fully functional version of the dystrophin gene into the body using a harmless virus, it doesn't mention anything about applicability of multi-exon skipping for DMD mutations in order to restore the open reading frame. However, in a recent interview with PPMD, Dr. Duan indicated that the CRISPR/Cas9 gene therapy that they've seen success with in dogs would also be successful for those DMD patients with a deletion of exons 46-50, and, indeed, other multi-exon mutations as well. (Go to page 13 for more details.)


Dr. Duan references this paper about the CRISPR/Cas9 technology:


Included in that link is the following that I wanted to draw your attention to. For our boys, "D" is the key:

(D) Schematic of multiplex sgRNA targets designed to delete the entire exon 45–55 region to address a variety of DMD patient mutations.

Figure 1

CRISPR/Cas9 targeting of the dystrophin gene

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