Star-shaped poly(l-lysine) with polyester bis-MPA dendritic core as potential degradable nano vectors for gene delivery
Smiljana Stefanovic, Katie McCormick, Sarinj Fattah, Ruiari Brannigan, Sally-Ann Cryan and Andreas Heise
Polymer Chemistry
School of Chemical Sciences
Abstract

Gene therapy has huge potential as a treatment for genetic conditions such as cystic fibrosis, alpha-1 antitrypsin deficiency, hemophilia, beta thalassemia, and sickle cell disease. It works by transferring genetic material to a patient’s cells To realise the immense possibilities presented by nucleic acid-based therapeutics, the development of effective and safe delivery vehicles is critical. Nucleic acids are large, negatively charged molecules and are rapidly cleared by circulating nucleases in the body. Suitable vectors must not only protect the gene cargo against damage but also efficiently deliver them intracellularly. One critical drawback of certain synthetic vectors is their dose dependent potential to damage human cells. While improved stability and easier scale-up is recognised as a benefit of these vectors, it is indeed surprising that their major advantage, the greater flexibility in structural design and the ability to easily modify them, has not been utilised to any large extend to overcome these drawbacks.