ArticleGene therapy2014
Targeting adipose tissue via systemic gene therapy.
Article in Gene therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
40 citing papers in PubMed, 61 citations in OpenAlex.
- AAV-mediated delivery of leptin but not adiponectin improves metabolic health in a pre-clinical mouse model of congenital generalised lipodystrophy.Gene therapy · 2026Article
- EctoShed: a novel gene delivery platform for functional analysis of adipocyte-shed proteoforms.BMC research notes · 2026Article
- MicroRNA-based targeting strategies enable AAV-mediated, tissue-selective gene expression in adipose tissue and skeletal muscle of mice.Molecular therapy. Advances · 2026Article
- The Role of Gene Therapy and RNA-Based Therapeutic Strategies in Diabetes.International journal of molecular sciences · 2025Review
- Dose-finding andMolecular therapy. Methods & clinical development · 2025Article
- Subcutaneous administration of adipose-tropic gene therapy for congenital leptin deficiency.Research square · 2025Article
- Review
- Pathogenetic therapeutic approaches for endocrine diseases based on antisense oligonucleotides and RNA-interference.Frontiers in endocrinology · 2025Review
- AAV-mediated co-expression of an immunogenic transgene plus PD-L1 enables sustained expression through immunological evasion.Scientific reports · 2024Article
- Gene Therapy Approach for Treatment of Obese Agouti Mice.International journal of molecular sciences · 2024Article
- Article
- Assessment of Adipocyte Transduction Using Different AAV Capsid Variants.Pharmaceuticals (Basel, Switzerland) · 2024Article
- Preclinical evaluation of tissue-selective gene therapies for congenital generalised lipodystrophy.Gene therapy · 2024Article
- Navigating Lipodystrophy: Insights from Laminopathies and Beyond.International journal of molecular sciences · 2024Review
- White-to-Beige and Back: Adipocyte Conversion and Transcriptional Reprogramming.Pharmaceuticals (Basel, Switzerland) · 2024Review
- Adipose tissue coregulates cognitive function.Science advances · 2023Article
- Direct Adeno-associated Viruses Injection of Murine Adipose Tissue.Bio-protocol · 2023Article
- AAV- based vector improvements unrelated to capsid protein modification.Frontiers in medicine · 2023Review
- AAV-Mediated Gene Delivery to Mouse Brown Adipose Tissue.Methods in molecular biology (Clifton, N.J.) · 2023Article
- Specific and efficient gene knockout and overexpression in mouse interscapular brown adipocytes in vivo.STAR protocols · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 2 institutions in 1 country.
Funding
Abstract
Adipose tissue has a critical role in energy and metabolic homeostasis, but it is challenging to adapt techniques to modulate adipose function in vivo. Here we develop an in vivo, systemic method of gene transfer specifically targeting adipose tissue using adeno-associated virus (AAV) vectors. We constructed AAV vectors containing cytomegalovirus promoter-regulated reporter genes, intravenously injected adult mice with vectors using multiple AAV serotypes, and determined that AAV2/8 best targeted adipose tissue. Altering vectors to contain adiponectin promoter/enhancer elements and liver-specific microRNA-122 target sites restricted reporter gene expression to adipose tissue. As proof of efficacy, the leptin gene was incorporated into the adipose-targeted expression vector, package into AAV2/8 and administered intravenously to 9- to 10-week-old ob/ob mice. Phenotypic changes were measured over an 8-week period. Leptin mRNA and protein were expressed in adipose and leptin protein was secreted into plasma. Mice responded with reversal of weight gain, decreased hyperinsulinemia and improved glucose tolerance. AAV2/8-mediated systemic delivery of an adipose-targeted expression vector can replace a gene lacking in adipose tissue and correct a mouse model of human disease, demonstrating experimental application and therapeutic potential in disorders of adipose.
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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.