ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Guanidinylated neomycin mediates heparan sulfate-dependent transport of active enzymes to lysosomes.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 32 citations in OpenAlex.
- R17 Drives Heparan Sulfate Clearance via Endolysosomal Trafficking.Israel journal of chemistry · 2026Article
- Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders.Advanced drug delivery reviews · 2023Review
- Comparison between Nanoparticle Encapsulation and Surface Loading for Lysosomal Enzyme Replacement Therapy.International journal of molecular sciences · 2022Article
- CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges.Frontiers in molecular biosciences · 2020Review
- Comprehensive review of chemical strategies for the preparation of new aminoglycosides and their biological activities.Chemical Society reviews · 2018Review
- Guanidinylated Neomycin Conjugation Enhances Intranasal Enzyme Replacement in the Brain.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Cellular uptake of modified aminoglycosides.The Journal of antibiotics · 2017Article
- Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.Advanced drug delivery reviews · 2017Review
- Polymyxins Facilitate Entry into Mammalian Cells.Chemical science · 2016Article
- On guanidinium and cellular uptake.The Journal of organic chemistry · 2014Article
- Macromolecular uptake of alkyl-chain-modified guanidinoglycoside molecular transporters.Chembiochem : a European journal of chemical biology · 2014Article
- Aggregation-mediated macromolecular uptake by a molecular transporter.ACS chemical biology · 2013Article
- Production of α-L-iduronidase in maize for the potential treatment of a human lysosomal storage disease.Nature communications · 2012Article
- Heparan sulfate proteoglycans.Cold Spring Harbor perspectives in biology · 2011Review
- Transport of arylsulfatase A across the blood-brain barrier in vitro.The Journal of biological chemistry · 2011Article
- Cooperative, heparan sulfate-dependent cellular uptake of dimeric guanidinoglycosides.Chembiochem : a European journal of chemical biology · 2010Article
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
Abstract
Guanidinylated neomycin (GNeo) can transport bioactive, high molecular weight cargo into the interior of cells in a process that depends on cell surface heparan sulfate proteoglycans. In this report, we show that GNeo-modified quantum dots bind to cell surface heparan sulfate, undergo endocytosis and eventually reach the lysosomal compartment. An N-hydroxysuccinimide activated ester of GNeo (GNeo-NHS) was prepared and conjugated to two lysosomal enzymes, beta-D-glucuronidase (GUS) and alpha-L-iduronidase. Conjugation did not interfere with enzyme activity and enabled binding of the enzymes to heparin-Sepharose and heparan sulfate on primary human fibroblasts. Cells lacking the corresponding lysosomal enzyme took up sufficient amounts of the conjugated enzymes to restore normal turnover of glycosaminoglycans. The high capacity of proteoglycan-mediated uptake suggests that this method of delivery might be used for enzyme replacement or introduction of foreign enzymes into cells.
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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.