ArticleProceedings of the National Academy of Sciences of the United States of America2004
Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2004. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 39 papers.
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Who cites it
39 citing papers in PubMed, 88 citations in OpenAlex.
- Sarcoma IL-12 overexpression facilitates NK cell immunomodulation.Scientific reports · 2021Article
- Anderson-Fabry Disease: From Endothelial Dysfunction to Emerging Therapies.Advances in pharmacological and pharmaceutical sciences · 2021Review
- Effect of lentiviral vector-packaged interleukin-18 gene on the malignant behavior of lung cancer.Experimental and therapeutic medicine · 2020Article
- Essential thrombocytosis attributed to JAK2-T875N germline mutation.International journal of hematology · 2019Article
- Inhibition of Mitochondrial Complex I Impairs Release of α-Galactosidase by Jurkat Cells.International journal of molecular sciences · 2019Article
- Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases.Genetics and molecular biology · 2019Article
- Neuraminidase 1 activates insulin receptor and reverses insulin resistance in obese mice.Molecular metabolism · 2018Article
- Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34Molecular therapy. Methods & clinical development · 2017Article
- Glycosphingolipid storage in Fabry mice extends beyond globotriaosylceramide and is affected by ABCB1 depletion.Future science OA · 2016Article
- Direct Lymph Node Vaccination of Lentivector/Prostate-Specific Antigen is Safe and Generates Tissue-Specific Responses in Rhesus Macaques.Biomedicines · 2016Article
- Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice.Gene therapy · 2013Article
- Engineering lentiviral vectors for modulation of dendritic cell apoptotic pathways.Virology journal · 2013Article
- Systemic ceramide accumulation leads to severe and varied pathological consequences.EMBO molecular medicine · 2013Article
- Development of a tridimensional microvascularized human skin substitute to study melanoma biology.Clinical & experimental metastasis · 2013Article
- MicroRNA-24 regulates cardiac fibrosis after myocardial infarction.Journal of cellular and molecular medicine · 2012Article
- Gene therapy approaches for lysosomal storage disease: next-generation treatment.Human gene therapy · 2012Review
- Lentivector transduction improves outcomes over transplantation of human HSCs alone in NOD/SCID/Fabry mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- In vivo gene transfer strategies to achieve partial correction of von Willebrand disease.Human gene therapy · 2012Article
- Bystander killing of malignant cells via the delivery of engineered thymidine-active deoxycytidine kinase for suicide gene therapy of cancer.Cancer gene therapy · 2012Article
- Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector.Human gene therapy · 2012Article
Corrections and comments
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Authors and funding
12 authors at 1 institution in 1 country.
Funding
Abstract
Successful therapy for many inherited disorders could be improved if the intervention were initiated early. This is especially true for lysosomal storage disorders. Earlier intervention may allow metabolic correction to occur before lipid buildup has irreversible consequences and/or before the immune system mounts limiting responses. We have been developing gene therapy to treat lysosomal storage disorders, especially Fabry disease. We describe studies directed toward metabolic correction in neonatal animals mediated by recombinant lentiviral vectors. To develop this method, we first injected a marking lentiviral vector that engineers expression of luciferase into the temporal vein of recipient neonatal animals. The use of a cooled charged-coupled device camera allowed us to track transgene expression over time in live animals. We observed intense luciferase expression in many tissues, including the brain, that did not diminish over 24 weeks. Next, we injected neonatal Fabry mice a single time with a therapeutic lentiviral vector engineered to express human alpha-galactosidase A. The injection procedure was well tolerated. We observed increased plasma levels of alpha-galactosidase A activity starting at our first plasma collection point (4 weeks). Levels of alpha-galactosidase A activity were found to be significantly elevated in many tissues even after 28 weeks. No immune response was observed against the corrective transgene product. Increased levels of enzyme activity also led to significant reduction of globotriaosylceramide in the liver, spleen, and heart. This approach provides a method to treat lysosomal storage disorders and other disorders before destructive manifestations occur.
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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.