A mixed-surface polyamidoamine dendrimer for in vitro and in vivo delivery of large plasmids

Ajit Sharma, Bhairavi Srinageshwar, Douglas Ray Swanson, Julien Pierre Andre Rossignol, Gary Dunbar, Maria Florendo, Brittany Clark, Kayla Johnson, Nikolas Munro, Sarah Peruzzaro, Aaron Antcliff, Melissa Andrews, Alexander Figacz

Research output: Contribution to journalArticlepeer-review

2 Scopus citations


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

Original languageEnglish
Article number619
Pages (from-to)1-15
Number of pages15
Issue number7
StatePublished - Jul 2020


  • Gene delivery
  • Glial cells
  • Large plasmid
  • Mixed-surface polyamidoamine dendrimers
  • Sox2


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