ArticleMolecular therapy : the journal of the American Society of Gene Therapy2017
Fatty Acid-Modified Gapmer Antisense Oligonucleotide and Serum Albumin Constructs for Pharmacokinetic Modulation.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
What it found
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Who cites it
20 citing papers in PubMed, 46 citations in OpenAlex.
- Albumin-Binding Fatty Acid-Modified Gapmer Antisense Oligonucleotides for Modulation of Pharmacokinetics.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Gapmers: Invention and Early History.Methods in molecular biology (Clifton, N.J.) · 2026Review
- Genetic therapies for neurological diseases.Pharmacological reviews · 2026Review
- Amphiphilic Oligonucleotide Derivatives-Promising Tools for Therapeutics.Pharmaceutics · 2024Review
- Improving DNA nanostructure stability: A review of the biomedical applications and approaches.International journal of biological macromolecules · 2024Review
- Structural optimization of siRNA conjugates for albumin binding achieves effective MCL1-directed cancer therapy.Nature communications · 2024Article
- A Modular Albumin-Oligonucleotide Biomolecular Assembly for Delivery of Antisense Therapeutics.Molecular pharmaceutics · 2024Article
- RNA nanostructures for targeted drug delivery and imaging.RNA biology · 2024Review
- The N-Myc-responsive lncRNA MILIP promotes DNA double-strand break repair through non-homologous end joining.Proceedings of the National Academy of Sciences of the United States of America · 2022Article
- Targeted Delivery of Immunostimulatory CpG Oligodeoxynucleotides to Antigen-Presenting Cells in Draining Lymph Nodes by Stearic Acid Modification and Nanostructurization.International journal of molecular sciences · 2022Article
- Safety Testing of an Antisense Oligonucleotide Intended for Pediatric Indications in the Juvenile Göttingen Minipig, including an Evaluation of the Ontogeny of Key Nucleases.Pharmaceutics · 2021Article
- Pharmacokinetics and Proceedings in Clinical Application of Nucleic Acid Therapeutics.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- Understanding In Vivo Fate of Nucleic Acid and Gene Medicines for the Rational Design of Drugs.Pharmaceutics · 2021Review
- The how and why of lncRNA function: An innate immune perspective.Biochimica et biophysica acta. Gene regulatory mechanisms · 2020Review
- Synthesis of Oligonucleotides Containing 2'-Molecules (Basel, Switzerland) · 2020Article
- Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle.Nucleic acids research · 2019Article
- Antisense-mediated splice intervention to treat human disease: the odyssey continues.F1000Research · 2019Review
- A self-assembled, modular nucleic acid-based nanoscaffold for multivalent theranostic medicine.Theranostics · 2019Article
- Harnessing albumin as a carrier for cancer therapies.Advanced drug delivery reviews · 2018Review
- Nanoparticle Technology: Having Impact, but Needing Further Optimization.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
Corrections and comments
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Delivery technologies are required for realizing the clinical potential of molecular medicines. This work presents an alternative technology to preformulated delivery systems by harnessing the natural transport properties of serum albumin using endogenous binding of gapmer antisense oligonucleotides (ASOs)/albumin constructs. We show by an electrophoretic mobility assay that fatty acid-modified gapmer and human serum albumin (HSA) can self-assemble into constructs that offer favorable pharmacokinetics. The interaction was dependent on fatty acid type (either palmitic or myristic acid), number, and position within the gapmer ASO sequence, as well as phosphorothioate (PS) backbone modifications. Binding correlated with increased blood circulation in mice (t
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.