UC Irvine

RNA based therapeutics have transformed biomedical research and attracted enormous attention due to the potential to treat uncurable genetic diseases. RNA based therapeutics involves the delivery of foreign RNA into cells to control gene expression, and ultimately alter protein expression. Through RNA therapeutics, a protein of interest can be produced through the introduction of specific messenger RNA (mRNA), transiently suppressed via RNA interference (RNAi), or permanently edited or deleted through the delivery of CRISPR-Cas9 gene editing machinery. However, the application of these groundbreaking technologies is hampered due to the lack of safe and effective delivery vehicles.

Chapter 1 provides a brief introduction to the background of gene therapy, along with challenges and considerations for developing effective gene delivery vehicles. Chapter 2 discusses a novel dipeptide functionalization strategy for developing new dendritic bolaamphiphile vectors for efficient siRNA transfection. Chapter 3 discusses the translation of the bolaamphiphile system into clinically relevant applications and in vitro delivery of siRNA to primary adipocyte and hepatocyte cells without the disruption of the underlying cellular function of the cells. Chapter 4 reports the delivery of CRISPR/Cas9 gene editing machinery via the codelivery of Cas9 mRNA and sgRNA in single polymeric nanoparticle.