CRISPR/Cas9 Gene Editing

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.)

Duan_2016_HGTCD.DMD_gene_therapy_interview.pdf

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

http://www.ncbi.nlm.nih.gov/pubmed/25692716

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

Figure 1