Ana Cubillo Alvarez, Dylan Maguire, Ruairí P Brannigan
{"title":"Synthetic-polymer-assisted antisense oligonucleotide delivery: targeted approaches for precision disease treatment.","authors":"Ana Cubillo Alvarez, Dylan Maguire, Ruairí P Brannigan","doi":"10.3762/bjnano.16.34","DOIUrl":null,"url":null,"abstract":"<p><p>This review explores the recent advancements in polymer-assisted delivery systems for antisense oligonucleotides (ASOs) and their potential in precision disease treatment. Synthetic polymers have shown significant promise in enhancing the delivery, stability, and therapeutic efficacy of ASOs by addressing key challenges such as cellular uptake, endosomal escape, and reducing cytotoxicity. The review highlights key studies from the past decade demonstrating how these polymers improve gene silencing efficiencies, particularly in cancer and neurodegenerative disease models. Despite the progress achieved, barriers such as immunogenicity, delivery limitations, and scalability still need to be overcome for broader clinical application. Emerging strategies, including stimuli-responsive polymers and advanced nanoparticle systems, offer potential solutions to these challenges. The review underscores the transformative potential of polymer-enhanced ASO delivery in personalised medicine, emphasising the importance of continued innovation to optimise ASO-based therapeutics for more precise and effective disease treatments.</p>","PeriodicalId":8802,"journal":{"name":"Beilstein Journal of Nanotechnology","volume":"16 ","pages":"435-463"},"PeriodicalIF":2.6000,"publicationDate":"2025-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11956074/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Beilstein Journal of Nanotechnology","FirstCategoryId":"88","ListUrlMain":"https://doi.org/10.3762/bjnano.16.34","RegionNum":4,"RegionCategory":"材料科学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/1/1 0:00:00","PubModel":"eCollection","JCR":"Q3","JCRName":"MATERIALS SCIENCE, MULTIDISCIPLINARY","Score":null,"Total":0}
Synthetic-polymer-assisted antisense oligonucleotide delivery: targeted approaches for precision disease treatment.
This review explores the recent advancements in polymer-assisted delivery systems for antisense oligonucleotides (ASOs) and their potential in precision disease treatment. Synthetic polymers have shown significant promise in enhancing the delivery, stability, and therapeutic efficacy of ASOs by addressing key challenges such as cellular uptake, endosomal escape, and reducing cytotoxicity. The review highlights key studies from the past decade demonstrating how these polymers improve gene silencing efficiencies, particularly in cancer and neurodegenerative disease models. Despite the progress achieved, barriers such as immunogenicity, delivery limitations, and scalability still need to be overcome for broader clinical application. Emerging strategies, including stimuli-responsive polymers and advanced nanoparticle systems, offer potential solutions to these challenges. The review underscores the transformative potential of polymer-enhanced ASO delivery in personalised medicine, emphasising the importance of continued innovation to optimise ASO-based therapeutics for more precise and effective disease treatments.
期刊介绍:
The Beilstein Journal of Nanotechnology is an international, peer-reviewed, Open Access journal. It provides a unique platform for rapid publication without any charges (free for author and reader) – Platinum Open Access. The content is freely accessible 365 days a year to any user worldwide. Articles are available online immediately upon publication and are publicly archived in all major repositories. In addition, it provides a platform for publishing thematic issues (theme-based collections of articles) on topical issues in nanoscience and nanotechnology.
The journal is published and completely funded by the Beilstein-Institut, a non-profit foundation located in Frankfurt am Main, Germany. The editor-in-chief is Professor Thomas Schimmel – Karlsruhe Institute of Technology. He is supported by more than 20 associate editors who are responsible for a particular subject area within the scope of the journal.