Structure-function assessment of mannosylated poly(beta-amino esters) upon targeted antigen presenting cell gene delivery and immune modulation
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Gene therapy is the manipulation of gene expression patterns in specific cells to treat genetic and pathological diseases. This manipulation is accomplished by the controlled introduction exogenous nucleic acids into target cells. Given the size and negative charge of these biomacromolecules, the delivery process is driven by the carrier vector, of which is dominated by the usage of viral vectors. Taking into account the limitations of viral vectors, nonviral alternatives have gained significant attention due to their flexible design, low cytotoxicity and immunogenicity, and their gene delivery efficacy. That stated, the field of nonviral vectors has been dominated by research dedicated to overcoming barriers in gene transfer. Unfortunately, these traditional nonviral vectors have failed to completely overcome the barriers required for clinical translation and thus, have failed to match the delivery outcomes of viral vector. This has consequently encouraged the development of new, more radical approaches that have the potential for higher clinical translation. With the use of contemporary characterization and physiological-mimicry tools, we are now capable of deciphering the intricate interplay of synthetic and biological mechanisms acting at different scales -- from the subcellular to the macroscopic -- endowing the ability to design and test strategies that will have improved translational capabilities.