ArticleGene therapy2012
Early gestational gene transfer with targeted ATP7B expression in the liver improves phenotype in a murine model of Wilson's disease.
Article in Gene therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
18 citing papers in PubMed, 40 citations in OpenAlex.
- Prevalence of pre-existing neutralizing antibodies to AAV5 and AAV8 in patients with Wilson's disease.Molecular therapy. Advances · 2026Article
- Recent advances in research on high-frequency mutations in the ATP7B gene associated with Wilson disease in the Chinese population: from genetic evolution to precision medicine.Frontiers in molecular biosciences · 2026Review
- Wilson Disease: Novel Diagnostic and Therapeutic Approaches.Seminars in liver disease · 2025Review
- Gene modification therapies for hereditary diseases in the fetus.Prenatal diagnosis · 2023Review
- Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells.PloS one · 2022Article
- Wilson Disease: Update on Pathophysiology and Treatment.Frontiers in cell and developmental biology · 2022Review
- A narrative review of in utero gene therapy: advances, challenges, and future considerations.Translational pediatrics · 2021Review
- The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism.Expert opinion on orphan drugs · 2020Article
- Biochemical Markers for the Diagnosis and Monitoring of Wilson Disease.The Clinical biochemist. Reviews · 2019Review
- Animal models of Wilson disease.Journal of neurochemistry · 2018Review
- Wilson's disease: Prospective developments towards new therapies.World journal of gastroenterology · 2017Review
- Targeted inactivation of copper transporter Atp7b in hepatocytes causes liver steatosis and obesity in mice.American journal of physiology. Gastrointestinal and liver physiology · 2017Article
- Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep.PloS one · 2017Article
- Bioavailable Trace Metals in Neurological Diseases.Current treatment options in neurology · 2016Review
- Wilson's disease and other neurological copper disorders.The Lancet. Neurology · 2015Review
- Modifying factors and phenotypic diversity in Wilson's disease.Annals of the New York Academy of Sciences · 2014Review
- Developmental stage determines efficiency of gene transfer to muscle satellite cells by in utero delivery of adeno-associated virus vector serotype 2/9.Molecular therapy. Methods & clinical development · 2014Article
- Biodistribution of Polymeric Nanoparticles following in utero Delivery to a Nonhuman Primate.Biomedicine hubArticle
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Authors and funding
8 authors at 3 institutions in 1 country.
Funding
Abstract
The ideal gene therapy for metabolical liver disorders would target hepatocytes before the onset of disease and be durable, non-toxic and non-immunogenic. Early gestational gene transfer can achieve such goals. Here, we demonstrate that prenatal gene transfer of human Atp7b reduces liver pathology and improves biochemical markers in Atp7b(-/-) mice, a murine model of Wilson's disease (WD). Following prenatal injection of lentivirus vector containing the human Atp7b gene under the transcriptional control of a liver-specific promoter, the full-length ATP7B was detectable in mouse livers for the entire duration of experiments (20 weeks after birth). In contrast to a marked pathology in non-injected animals, livers from age-matched treated mice consistently demonstrated normal gross and histological morphology. Hepatic copper content was decreased in the majority of treated mice, although remaining copper levels varied. Improvement of hepatic copper metabolism was further apparent from the presence of copper-bound ceruloplasmin in the sera and normalization of the mRNA levels for HMG CoA-reductase. With this approach, the complete loss of copper transport function can be ameliorated, as evident from phenotypical improvement in treated Atp7b(-/-) mice. This study provides proof of principle for in utero gene therapy in WD and other liver-based enzyme deficiencies.
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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.