Recent innovations in nonviral delivery approaches for gene therapies

Developing vectors for safe, effective and targeted delivery could accelerate gene therapy innovation. Genetic medicines have transformed the way we treat some diseases, from inherited blindness to blood cancers, and – in some cases – all in a single, curative injection. With the ever-expanding menu of gene modification tools such as CRISPR, messenger RNAs, antisense oligonucleotides, microRNAs, and more, research has now shifted to how these tools can be successfully applied in treating diseases in a manner that is both safe and effective. Several FDA-approved gene therapies use viral vectors such as adeno-associated viruses (AAVs) to deliver genes into cells...