ArticleProceedings of the National Academy of Sciences of the United States of America2008
Aptamer-based endocytosis of a lysosomal enzyme.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 68 papers.
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Who cites it
68 citing papers in PubMed, 159 citations in OpenAlex.
- Immunoaffinity-Mimetic Assembly of Peptide-Aptamer Conjugates and Stem Cell-Derived Exosomes into Hierarchical Microgels for Spinal Cord Injury Repair.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Navigating the next frontier in biomedicine: breakthroughs and insights in nucleic acid therapeutics.Chemical science · 2026Review
- Triple-Targeted DNA Nanozyme Executes Disulfidptosis for Glioma Elimination.Theranostics · 2026Article
- Aptamers in Drug Delivery Development.Materials today (Kidlington, England) · 2025Article
- Nucleic acid aptamers in orthopedic diseases: promising therapeutic agents for bone disorders.Bone research · 2025Review
- Aptamer Technologies in Neuroscience, Neuro-Diagnostics and Neuro-Medicine Development.Molecules (Basel, Switzerland) · 2024Review
- Exploring the Potential of Aptamers in Targeting Neuroinflammation and Neurodegenerative Disorders: Opportunities and Challenges.International journal of molecular sciences · 2023Review
- Transferrin Aptamers Increase theBioconjugate chemistry · 2022Article
- Article
- Nanoparticle-Guided Brain Drug Delivery: Expanding the Therapeutic Approach to Neurodegenerative Diseases.Pharmaceutics · 2021Review
- The biological applications of DNA nanomaterials: current challenges and future directions.Signal transduction and targeted therapy · 2021Review
- A DNA Aptameric Ligand of Human Transferrin Receptor Generated by Cell-SELEX.International journal of molecular sciences · 2021Article
- Physiological and Pathological Factors Affecting Drug Delivery to the Brain by Nanoparticles.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2021Review
- Overcoming Barriers for siRNA Therapeutics: From Bench to Bedside.Pharmaceuticals (Basel, Switzerland) · 2020Review
- Selection of High-Affinity RNA Aptamers That Distinguish between Doxycycline and Tetracycline.Biochemistry · 2020Article
- Non-Invasive Delivery of Therapeutics into the Brain: The Potential of Aptamers for Targeted Delivery.Biomedicines · 2020Review
- Targeted Delivery of C/EBPα-saRNA by RNA Aptamers Shows Anti-tumor Effects in a Mouse Model of Advanced PDAC.Molecular therapy. Nucleic acids · 2019Article
- Development of Novel antimiRzymes for Targeted Inhibition of miR-21 Expression in Solid Cancer Cells.Molecules (Basel, Switzerland) · 2019Article
- Brassica rapa hairy root based expression system leads to the production of highly homogenous and reproducible profiles of recombinant human alpha-L-iduronidase.Plant biotechnology journal · 2019Article
- Aptamer-miR-34c Conjugate Affects Cell Proliferation of Non-Small-Cell Lung Cancer Cells.Molecular therapy. Nucleic acids · 2018Article
8 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 1 institution in 1 country.
Funding
Abstract
Enzyme replacement therapy for lysosomal storage diseases is currently based on endocytosis of lysosomal enzymes via the mannose or mannose 6-phosphate receptors. We are developing a technology for endocytosis of lysosomal enzymes that depends on generic, chemically conjugated reagents. These reagents are aptamers (single-stranded nucleic acid molecules) selected to bind to the extracellular domain of the mouse transferrin receptor. After selection, an RNA aptamer and a DNA aptamer were modified with biotin and linked to dye-labeled streptavidin for detection by confocal microscopy. Aptamer-streptavidin conjugates showed saturable uptake into mouse fibroblasts (Ltk(-) cells), which could be inhibited by an excess of free aptamer but not by tRNA, calf thymus DNA, or transferrin. The RNA aptamer-streptavidin conjugate was mouse-specific, as human cells (293T) did not take it up unless first transfected with the mouse transferrin receptor. Some streptavidin separated from the recycling pathway of transferrin and colocalized with lysosomes. After characterization in the model system, the DNA aptamer was conjugated to a lysosomal enzyme, alpha-l-iduronidase, from which mannose 6-phosphate had been removed. The aptamer had been modified by attachment of terminal glycerol for oxidation by periodate and reaction of the resulting aldehyde with amino groups on the protein. Dephospho-alpha-L-iduronidase-aptamer conjugate was taken up in saturable manner by alpha-L-iduronidase-deficient mouse fibroblasts, with half-maximal uptake estimated as 1.6 nM. Endocytosed enzyme-aptamer conjugate corrected glycosaminoglycan accumulation, indicating that it reached lysosomes and was functional in those organelles. Both uptake and correction were inhibited by unconjugated aptamer, confirming the role of the aptamer in receptor-mediated endocytosis.
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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.