Cationic, amphiphilic copolymer micelles as nucleic acid carriers for enhanced transfection in rat spinal cord

Spinal cord injuries often result in permanent motor and sensory deficits due to the limited regenerative capacity of the adult central nervous system. Nucleic acid-based therapies are a promising strategy for delivering bioactive molecules that can promote axonal regeneration. The study introduces a novel nonviral vector, poly (lactide-co-glycolide)-graft-polyethylenimine (PgP) micelle, capable of efficiently delivering plasmid DNA (pDNA) and siRNA in the presence of serum. The developed PgP copolymer enhances transfection and reduces cytotoxicity in neural cells both in vitro and in vivo, suggesting its potential as a nonviral vector for therapeutic nucleic acid delivery aimed at neural regeneration.

• 1
  PgP micelles efficiently transfect pDNA and siRNA in the presence of 10% serum in neuroglioma (C6) cells, neuroblastoma (B35) cells, and primary E8 chick forebrain neurons (CFN).
• 2
  PgP provides high-level transgene expression in the rat spinal cord in vivo, substantially greater than that attained with bPEI.
• 3
  PgP/pβ-Gal achieved substantial transfection in the injection site and surrounding neural tissue that was substantially higher than bPEI/pβ-Gal or naked pβ-Gal controls.