ArticleMolecular therapy : the journal of the American Society of Gene Therapy2005
Defining the pathway for Tat-mediated delivery of beta-glucuronidase in cultured cells and MPS VII mice.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2005. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
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Who cites it
26 citing papers in PubMed, 43 citations in OpenAlex.
- Cracking the Blood-Brain Barrier Code: Rational Nanomaterial Design for Next-Generation Neurological Therapies.Pharmaceutics · 2025Review
- A Historical Review of Brain Drug Delivery.Pharmaceutics · 2022Review
- Brain Delivery of Nanomedicines: Trojan Horse Liposomes for Plasmid DNA Gene Therapy of the Brain.Frontiers in medical technology · 2020Review
- Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis.Cellular and molecular life sciences : CMLS · 2019Review
- Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Guanidinylated Neomycin Conjugation Enhances Intranasal Enzyme Replacement in the Brain.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.Advanced drug delivery reviews · 2017Review
- Protein Delivery of an Artificial Transcription Factor Restores Widespread Ube3a Expression in an Angelman Syndrome Mouse Brain.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- Corrective GUSB transfer to the canine mucopolysaccharidosis VII cornea using a helper-dependent canine adenovirus vector.Journal of controlled release : official journal of the Controlled Release Society · 2014Article
- Corrective GUSB transfer to the canine mucopolysaccharidosis VII brain.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- HIV Tat Domain Improves Cross-correction of Human Galactocerebrosidase in a Gene- and Flanking Sequence-dependent Manner.Molecular therapy. Nucleic acids · 2013Article
- Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders.Drug delivery and translational research · 2012Article
- Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy.Human molecular genetics · 2010Article
- Guanidinylated neomycin mediates heparan sulfate-dependent transport of active enzymes to lysosomes.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Enhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Fluorescent sterols monitor cell penetrating peptide Pep-1 mediated uptake and intracellular targeting of cargo protein in living cells.Biochimica et biophysica acta · 2009Article
- Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood-brain barrier in the newborn mouse.Molecular therapy : the journal of the American Society of Gene Therapy · 2008Article
- Infused Fc-tagged beta-glucuronidase crosses the placenta and produces clearance of storage in utero in mucopolysaccharidosis VII mice.Proceedings of the National Academy of Sciences of the United States of America · 2008Article
- Genetic incorporation of the protein transduction domain of Tat into Ad5 fiber enhances gene transfer efficacy.Virology journal · 2007Article
- New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy.Human genetics · 2007Review
Corrections and comments
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Authors and funding
11 authors at 4 institutions in 2 countries.
Funding
Abstract
We used recombinant forms of human beta-glucuronidase (GUS) purified from secretions from stably transfected CHO cells to compare the native enzyme to a GUS-Tat C-terminal fusion protein containing the 11-amino-acid HIV Tat protein transduction domain for: (1) susceptibility to endocytosis by cultured cells, (2) rate of clearance following intravenous infusion, and (3) tissue distribution and effectiveness in clearing lysosomal storage following infusion in the MPS VII mouse. We found: (1) Native GUS was more efficiently taken up by cultured human fibroblasts and its endocytosis was exclusively mediated by the M6P receptor. The GUS-Tat fusion protein showed only 30-50% as much M6P-receptor-mediated uptake, but also was taken up by adsorptive endocytosis through binding of the positively charged Tat peptide to cell surface proteoglycans. (2) GUS-Tat was less rapidly cleared from the circulation in the rat (t(1/2) = 13 min vs 7 min). (3) Delivery to most tissues of the MPS VII mouse was similar, but GUS-Tat was more efficiently delivered to kidney. Histology showed that GUS-Tat more efficiently reduced storage in renal tubules, retina, and bone. These studies demonstrate that Tat modification can extend the range of tissues corrected by infused enzyme.
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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.