Research

Hormonal

Single-dose AAV-mediated liver-specific FGF21 gene therapy reverses diet-induced obesity, hepatic steatosis, and insulin resistance in mice through sustained elevation of circulating FGF21 and increased energy expenditure.

This research suggests that a single injection of a viral vector delivering the FGF21 gene to the liver can sustainably reverse obesity and insulin resistance in mice by boosting energy expenditure. While promising, this is preclinical data; human applications are not yet established.

GoodSupportsHIGH confidence
Here, we demonstrate for the first time that a single administration of AAV vectors encoding FGF21 enabled a long-lasting increase in FGF21 levels in circulation, which resulted in sustained counteraction of obesity, hepatic steatosis, and insulin resistance in two different models of obesity and T2D, the HFD-fed mouse and the ob/ob mouse.
Verónica Jiménez et al. · EMBO Molecular Medicine · 2018

Why this rating

High-quality preclinical data in multiple mouse models with long-term follow-up (>1 year), but lacks human clinical trial data.

Source

FGF21 gene therapy as treatment for obesity and insulin resistance

Verónica Jiménez et al. · EMBO Molecular Medicine · 2018

DOI 10.15252/emmm.201708791

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DOI resolved against Crossref · corpus check 2026-06-10

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