Sangamo In Vivo Gene Editing for Enzyme/Protein Replacement Therapy
posted on
Nov 17, 2017 10:42AM
Thanks to JD on the Stockhouse board for bringing this milestone to our attention. Sangamo is using zinc-finger nuclease gene editing tools with an adeno-associated viral (AAV) vector delivery vehcile in vivo in human trials to correct enzyme deficiency diseases such as Hunter syndrome (MPS II). In short, this allows them to genetically edit some of the liver cells and replace the albumin genetic locus with that of the IDS gene that is mutated/deficient in MPS II. The end result is turning the liver into a IDS generating factory to give long-term enzyme replacement w/o regular infusion of recombinant IDS enzyme.
How does this tie into biOasis? Well, regardless of the liver making the IDS enzyme or the patient receiving infusion of IDS enzyme, the blood brain barrier is an obstactle for getting the enzyme into the brain. The xB3 (formerly Transcendpep, Mtfp) could be a key for augmenting this Sangamo approach to enable sufficient enzyme replacement to the brain.
In vivo genome editing of the albumin locus as a platform for protein replacement therapy.