A major challenge in gene therapy is the development of safe and efficient methods of gene delivery to target cells. Currently, the most common methods of gene delivery utilize viral vectors and synthetic transfer vehicles. Although viral vectors display high transfer efficiency, they are plagued by the induction of adverse immune responses, limitations on payload size and high production costs. While synthetic vehicles are generally immunologically neutral, these reagents are handicapped by low efficiency and toxicity. Dr. Clifton Ragsdale and Dr. Luping Yu of The University of Chicago have developed a novel class of gene delivery polymers that overcome these shortcomings. They have constructed a large library of poly(β-aminosulfonamide) (PBAS) polymers and using high throughput in vitro and in vivo screening techniques have identified several PBAS polymers that greatly outperform currently available synthetic delivery vehicles in vitro and in vivo. Furthermore, the stability of these polymers allows for the attachment of targeting moieties to produce delivery vehicles that transport their payloads to specific subsets of cells in vivo. These polymers not only are a huge step forward in the in vitro and in vivo transfection of cells for use in research, but also have the potential to revolutionize the field of gene therapy.

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