Abstract
Polymers Tantalizing promise, largely unfulfilled, haunts gene therapy for human disease. Among several barriers to success, efficient gene delivery to targeted cells remains daunting. Zhou et al. broke one barrier through the synthesis of a new class of delivery vector. The pathway results in the synthesis of highly branched poly(β-amino ester) (HPAE), using a one-pot Michael addition. The transfection efficiency of HPAE over existing vectors improved by as much as three orders of magnitude. Gene delivery by HPAE was able to correct a genetic defect in a recessive dystrophic epidermolysis bullosa graft mouse model. Sci. Adv. 10.1126.sciadv.1600102 (2016).
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