Going into Warp Drive in Gene Therapy - In Vivo Gene Editing


One the problems with writing a book is that it tends to occupy most of your time and you tend to ignore  work that is not value-added for the current work.  While one needs focus for writing, this can mean missing some really important stuff.    So it was a bit of surprise today when I read an article by Sara Reardon in Nature entitled "Gene-editing wave hits the clinic. (Reardon, S. Gene-editing wave hits the clinic,  Nature 527(Issue Number 7577), 146-147, 2015).   While I had noticed that gene editing techniques such as crispr cas9 had potential for in vivo application, I was not aware of the progress being made by various investigators.  Reardon describes work by  Sagamo Biosciences (Richmond, California) on use of in vivo gene editing technology to correct the coagulation defect in hemophilia B. This work used a gene editing technique directed at the albumin gene locus to improve expression (Sharma, R., Anguela, X.M., Doyon, Y.., et al., In vivo genome editing of the albumin locus as a platform for protein replacement therapy, Blood 126(18), 1777-1784, 2015).  This work must create some sleepless nights for those companies developing new products for hemophilia A and hemophilia B.  There will be marketing issues for gene therapy which may be a service and a product but such can be solved by thoughtful people with considerable experience.  This would be  similar to IVIG before there was back to the future with SC administration of product.  Reardon had another article in the same issue of Nature on the use of gene editing for use in pig organs for xenotransplantation (Reardon, S., New life for pig organs, Nature 527(Issue number 7577), 152-154, 2015).