Gene therapy’s new hope: A neuron-targeting virus is saving infant lives

An amazing article highlighting the therapeutic potential of treating certain neurological disorders using adeno-associated viral (AAV) vectors. Certain AAVs, like AAV9, really seem to home in on neurons and allow for AAV-mediated gene expression in sufficient quantities in the brain to correct genetic deficiencies. Importantly, the AAV is delivered intravenously and a sufficient amount gets delivered to the brain.

Of course, this AAV technology isn't amenable to all type of therapies and therefore xB3 is a much more versatile delivery vehicle. But this AAV9 news definitely gives hope to treating diseases that require restoring neuronal/brain synthesis of a particular gene product/protein. Interestingly, the article references an ongoing trial using an AAV9 approach to restore the enzyme that is deficient in the lysosomal storage disease (LSD) Sanfilippo type A syndrome (MPS III). So there may be some competition for trojan horse approaches like biOasis and the xB3 peptide for treating LSDs.

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