Drug delivery to the brain is highly hindered by the presence of the bloodâ??brain barrier\n(BBB), which prevents the entry of many potential drugs/biomolecules into the brain. One of the\ncurrent strategies to achieve gene therapy for neurodegenerative diseases involves direct injection of\na viral vector into the brain. There are various disadvantages of viral vectors, including limitations of\ncargo size and safety concerns. Nanomolecules, such as dendrimers, serve as an excellent alternative\nto viral delivery. In this study, as proof-of-concept, we used a surface-modified dendrimer complex\nand delivered large plasmids to cells in vitro and in vivo in healthy rats via intracranial injection.\nThe dendrimers were biodegradable by chemicals found within cells and toxicity assays revealed\nthat the modified dendrimers were much less toxic than unmodified amine-surface dendrimers.\nAs mentioned in our previous publication, these dendrimers with appropriately modified surfaces are\nsafe, can deliver large plasmids to the brain, and can overcome the cargo size limitations associated\nwith viral vectors. The biocompatibility of this dendritic nanomolecule and the ability to finely tune\nits surface chemistry provides a gene delivery system that could facilitate future in vivo cellular\nreprograming and other gene therapies.
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