Monash University researchers have developed a versatile nanobody-antibody system to enhance mRNA lipid nanoparticle (LNP) targeting to specific organs, improving therapeutic precision without modifying antibodies themselves. Additionally, gene therapy research demonstrates promising breakthroughs, including cDNA insertion techniques for chronic granulomatous disease and strategies to treat juvenile neurodegenerative CLN3 disease’s neuromuscular symptoms. These developments underline ongoing efforts in improving delivery systems and therapeutic safety in gene and RNA therapies.