Synthetic-polymer-assisted antisense oligonucleotide delivery: targeted approaches for precision disease treatment

• Ana Cubillo Alvarez,
• Dylan Maguire and
• Ruairí P. Brannigan

Beilstein J. Nanotechnol. 2025, 16, 435–463, doi:10.3762/bjnano.16.34

• optimise ASO-based therapeutics for more precise and effective disease treatments. Keywords: antisense oligonucleotides; enhanced delivery; gene transfection; intracellular uptake; locked nucleic acid (LNA); nanoparticles; peptide nucleic acid (PNA); personalised therapy; phosphorodiamidate morpholino

• oligomer (PMO); phosphorothioate (PS); polyplexes; ribose substitutions; small interfering RNA (siRNA); synthetic polymers; tricyclo-DNA (tcDNA); Introduction The development and use of personalised therapies tailored to individual patients have emerged as a powerful strategy for treating various