Adenovirus associated virus (AAV) vectors are being increasingly used for gene therapy because they are not pathogenic in humans and persist for long periods in certain cell types. Currently 120 gene delivery clinical trials with these vectors are in progress, and two have been approved: Luxturna to treat a rare form of blindness, and another for the treatment of spinal muscular atrophy. Despite these successes, improvements of the efficiency of gene delivery by these vectors are needed. In silico reconstructions of putative ancestors of AAV has led to the development of a new vector that is efficiently expressed in multiple cell types.
[Read more…] about An ancestral vector improves on this year’s model