Efficient neuronal targeting and transfection using RVG and transferrin-conjugated liposomes

Bruna dos Santos Rodrigues, Sanjay Arora, Takahisa Kanekiyo, Jagdish Singh

Research output: Contribution to journalArticlepeer-review

5 Scopus citations


Effective transport of therapeutic nucleic acid to brain has been a challenge for the success of gene therapy for treating brain diseases. In this study, we proposed liposomal nanoparticles modified with brain targeting ligandsfor active brain targeting with enhanced BBB permeation and delivery of genes to brain. We targeted transferrin and nicotinic acetylcholine receptors by conjugating transferrin (Tf) and rabies virus glycoprotein (RVG) peptide to surface of liposomes. Liposomal formulations showed homogeneous particle size and ability to protect plasmid DNA against enzymatic degradation. These nanoparticles were internalized by brain endothelial cells, astrocytes and primary neuronal cells through energy-dependent endocytosis pathways. RVG-Tf coupled liposomes showed superior ability to transfect cells compared to liposomes without surface modification or single modification. Characterization of permeability through blood brain barrier (BBB) and functionality of designed liposomes were performed using an in vitro triple co-culture BBB model. Liposome-RVG-Tf efficiently translocated across in vitro BBB model and, consecutively, transfected primary neuronal cells. Notably, brain-targeted liposomes promoted in vivo BBB permeation. These studies suggest that modifications of liposomes with brain-targeting ligands are a promising strategy for delivery of genes to brain.

Original languageEnglish (US)
Article number146738
JournalBrain Research
StatePublished - May 1 2020


  • Brain
  • Gene therapy
  • Liposome
  • RVG
  • Transferrin

ASJC Scopus subject areas

  • General Neuroscience
  • Molecular Biology
  • Clinical Neurology
  • Developmental Biology


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