ArticleMolecular therapy. Nucleic acids2025
A novel gene therapy platform for the treatment of type 2 diabetes and obesity.
Article in Molecular therapy. Nucleic acids, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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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
1 citing paper in PubMed.
- Low dose systemic AAV-exendin-4 gene therapy for Prader-Willi syndrome and dietary obesity.Molecular therapy. Advances · 2026Article
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10 authors.
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No grant is acknowledged in the PubMed record.
Abstract
Diabetes and obesity are a growing healthcare and socioeconomic crisis of unprecedented proportions. Driven in part by the westernization of diets around the world, the prevalence of individuals with type 2 diabetes alone is projected to exceed 1.3 billion by 2050. Glucagon-like peptide 1 (GLP1) receptor agonists offer transformative therapeutic potential but are challenged by gastrointestinal side effects due to pharmacokinetic spikes from repeated injections, resulting in discontinuation rates which approach 70% by the first anniversary of treatment initiation. To overcome the repeat-injection nature of current GLP1 therapies, we developed a novel, subcutaneously injectable, lipid nanoparticle-based DNA delivery system to administer and express exendin 4 (EX4) or a modified natural GLP1 peptide in obese diabetic mice. The treatment was well tolerated, durable, and the transgene remained localized to the subcutaneous area of injection for over 6 months. The expressed EX4 and GLP1 promoted weight loss and decreased food intake, while reducing insulin resistance and improving glycemic control. Changes in the transcriptomic profile indicated that efficacy was mediated via the GLP1 receptor pathway, and blood-based biomarkers of liver and pancreatic function, systemic inflammation, and muscle injury confirmed that the treatment was systemically well tolerated.
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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.