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Oligonucleotide Therapeutics & Delivery 3

Duplicate DOIs are merged. This is a selected collection, not an exhaustive bibliography.

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Reviews & perspectives
AAPS Open

Perspective maps oligonucleotide conjugation strategies beyond liver targeting

Ya Ying Zheng; Yonger Xue; Xucheng Hou; Yizhou Dong

An AAPS Open perspective surveys conjugation strategies for antisense oligonucleotides and siRNAs, including small-molecule ligands, cell-penetrating peptides, antibodies, and aptamers. The abstract contrasts clinically established GalNAc delivery with emerging approaches to extrahepatic tissues and discusses translation challenges. This is a synthesis of delivery platforms and clinical progress, rather than a new experimental or clinical efficacy study.

Oligonucleotide Therapeutics & Delivery
Read guide →Original paper ↗

DOI: 10.1186/s41120-026-00209-8

Original research
Nucleic Acid Therapeutics

GuNA-modified antisense gapmers show prolonged activity in mouse brain

Takaaki Kawanobe; Ajaya Ram Shrestha; Hideaki Tomita

Based on the PubMed abstract, researchers combined guanidine-bridged nucleic acid and MOE modifications in antisense gapmers, reporting improved knockdown in laboratory and animal experiments. Activity persisted beyond 24 weeks in mouse brain after intracerebroventricular administration. The study also examined immune responses and toxicity; these preclinical results do not establish human safety or efficacy.

Oligonucleotide Therapeutics & Delivery
Read guide →Original paper ↗

DOI: 10.1177/21593337261502560

Reviews & perspectives
Pharmacological Reviews

Pharmaceutical perspectives on oligonucleotide therapeutics and delivery systems

Dalton W Staller; Flobater I Gawargi; Sanjali S Panigrahi; Paras K Mishra; Ram I Mahato

This review examines RNA species, structure and function, chemical modifications, and delivery systems in the context of therapeutic development. It connects molecular design with stability and distribution and discusses clinical applications available at the time of writing. Its historical approval overview should not be interpreted as a current, exhaustive drug list.

Oligonucleotide Therapeutics & Delivery
Read guide →Original paper ↗

DOI: 10.1016/j.pharmr.2025.100065

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