Development of Degradable Cationic Shell Crosslinked Nanoparticles toward Efficient Transfection of Nucleic Acids

摘要

Gene therapy is a medical technique that is used to repair genetic defects by introducing a normal or functional gene to replace or correct a faulty gene. Although viral-based vectors are known for their high transfection efficiencies, the large number of drawbacks including induction of an immune response against the viral proteins, possible recombination with wild-type viruses and challenges in large-scale production has drawn much attention away from the viral vectors toward synthetic (non-viral based) gene delivery systems that allow for safer administration, ease of preparation, versatility in tailoring their chemical and biological properties, and cost effectiveness.