AAV-deliverable base editors; plus a newborn genetic disease screening tool and more

Clinical utility of base editors is limited by their inability to fit into adeno-associated viral (AAV) vectors. Two groups developed smaller versions that fit into a single vector.

Led by David Liu, Broad Institute of MIT and Harvard researchers revealed condensed adenine base editors that outperformed efficiencies of dual-AAV approaches in heart and skeletal muscle, possibly allowing a reduction in the dose required for the desired level of editing. “These findings suggest that a single-AAV system may be especially preferable when targeting non-liver tissues, or when toxicity limits AAV dosage,” the authors wrote in the Nature Biomedical Engineering article...