ArticleDrug design, development and therapy2012
Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy.
Article in Drug design, development and therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 1 of them a synthesis that pooled it.
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Who cites it
10 citing papers in PubMed, 1 synthesis or guideline pooled it, 28 citations in OpenAlex.
- Fabry disease cardiomyopathy: A state-of-the-art review.Progress in cardiovascular diseasesPooled it
- Galactomannan-Decorated Lipidic Nanocarrier for Gene Supplementation Therapy in Fabry Disease.Nanomaterials (Basel, Switzerland) · 2022Article
- Fabry disease: Mechanism and therapeutics strategies.Frontiers in pharmacology · 2022Review
- The New Pharmacological Chaperones PBXs Increase α-Galactosidase A Activity in Fabry Disease Cellular Models.Biomolecules · 2021Article
- α-Galactosidase A Augmentation by Non-Viral Gene Therapy: Evaluation in Fabry Disease Mice.Pharmaceutics · 2021Article
- Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A.Molecular therapy. Methods & clinical development · 2021Article
- New drugs for the treatment of Anderson-Fabry disease.Journal of nephrology · 2021Review
- Anderson-Fabry cardiomyopathy: prevalence, pathophysiology, diagnosis and treatment.Heart failure reviews · 2015Review
- Lipid nanoparticles as carriers for RNAi against viral infections: current status and future perspectives.BioMed research international · 2014Review
- Development of nucleic acid vaccines: use of self-amplifying RNA in lipid nanoparticles.International journal of nanomedicine · 2014Review
Corrections and comments
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeGene-mediated enzyme replacement is a reasonable and highly promising approach for the treatment of Fabry disease (FD). The objective of the present study was to demonstrate the potential applications of solid lipid nanoparticle (SLN)-based nonviral vectors for the treatment of FD.
methodsSLNs containing the pR-M10-αGal A plasmid that encodes the α-Galactosidase A (α-Gal A) enzyme were prepared and their in vitro transfection efficacy was studied in Hep G2 cells. We also studied the cellular uptake of the vectors and the intracellular disposition of the plasmid.
resultsThe enzymatic activity of the cells treated with the vectors increased significantly relative to the untreated cells, regardless of the formulation assayed. When the SLNs were prepared with protamine or dextran and protamine, the activity of the α-Gal A enzyme by the transfected Hep G2 cells increased up to 12-fold compared to that of untreated cells.
conclusionWith this work we have revealed in Hep G2 cells the ability of a multicomponent system based on SLNs to act as efficient nonviral vectors to potentially correct low α-Gal A activity levels in FD with gene therapy.
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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.