ArticleJournal of biomedical science2010
Characterization of Fabry mice treated with recombinant adeno-associated virus 2/8-mediated gene transfer.
Article in Journal of biomedical science, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed, 23 citations in OpenAlex.
- Reversing Pathology in an Aggravated Fabry Mouse Model Using Low-Dose Engineered Human Alpha-Galactosidase A AAV Gene Therapy.Biomedicines · 2025Article
- Fabry disease exacerbates renal interstitial fibrosis after unilateral ureteral obstruction via impaired autophagy and enhanced apoptosis.Kidney research and clinical practice · 2021Article
- Anderson-Fabry Disease: From Endothelial Dysfunction to Emerging Therapies.Advances in pharmacological and pharmaceutical sciences · 2021Review
- Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases.Genetics and molecular biology · 2019Article
- Anderson-Fabry disease in heart failure.Biophysical reviews · 2018Review
- Depletion of globosides and isoglobosides fully reverts the morphologic phenotype of Fabry disease.Cell and tissue research · 2014Article
- Enhanced sialylation and in vivo efficacy of recombinant human α-galactosidase through in vitro glycosylation.BMB reports · 2013Article
- Article
- Sex differences of urinary and kidney globotriaosylceramide and lyso-globotriaosylceramide in Fabry mice.Journal of lipid research · 2011Article
- Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiency.Human gene therapy · 2011Article
- Clarifying lysosomal storage diseases.Trends in neurosciences · 2011Review
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundEnzyme replacement therapy (ERT) with alpha-galactosidase A (alpha-Gal A) is currently the most effective therapeutic strategy for patients with Fabry disease, a lysosomal storage disease. However, ERT has limitations of a short half-life, requirement for frequent administration, and limited efficacy for patients with renal failure. Therefore, we investigated the efficacy of recombinant adeno-associated virus (rAAV) vector-mediated gene therapy for a Fabry disease mouse model and compared it with that of ERT.
methodsA pseudotyped rAAV2/8 vector encoding alpha-Gal A cDNA (rAAV2/8-hAGA) was prepared and injected into 18-week-old male Fabry mice through the tail vein. The alpha-Gal A expression level and globotriaosylceramide (Gb3) levels in the Fabry mice were examined and compared with Fabry mice with ERT. Immunohistochemical and ultrastructural studies were conducted.
resultsTreatment of Fabry mice with rAAV2/8-hAGA resulted in the clearance of accumulated Gb3 in tissues such as liver, spleen, kidney, heart, and brain with concomitant elevation of alpha-Gal A enzyme activity. Enzyme activity was elevated for up to 60 weeks. In addition, expression of the alpha-Gal A protein was identified in the presence of rAAV2/8-hAGA at 6, 12, and 24 weeks after treatment. alpha-Gal A activity was significantly higher in the mice treated with rAAV2/8-hAGA than in Fabry mice that received ERT. Along with higher alpha-Gal A activity in the kidney of the Fabry mice treated with gene therapy, immunohistochemical studies showed more alpha-Gal A expression in the proximal tubules and glomerulus, and less Gb3 deposition in Fabry mice treated with this gene therapy than in mice given ERT. The alpha-gal A gene transfer significantly reduced the accumulation of Gb3 in the tubules and podocytes of the kidney. Electron microscopic analysis of the kidneys of Fabry mice also showed that gene therapy was more effective than ERT.
conclusionsThe rAAV2/8-hAGA mediated alpha-Gal A gene therapy provided improved efficiency over ERT in the Fabry disease mouse model. Furthermore, rAAV2/8-hAGA-mediated expression showed a greater effect in the kidney than ERT.
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