1324-DNA TFO Liposomes for Pulmonary Fibrosis Gene Therapy

The paper details a scientific study published in Cell Reports Medicine regarding a novel gene therapy designed to treat pulmonary fibrosis. Researchers developed a targeted DNA triplex-forming oligonucleotide (TFO) liposome, known as tLipo-TFO1, which specifically targets fibrotic lung cells using an isoDGR peptide that binds to $\alpha$v$\beta$6 integrin receptors. By forming a DNA triplex at the Serpine1 gene site, this therapy effectively suppresses the production of plasminogen activator inhibitor 1 (PAI-1). Reducing PAI-1 levels activates the fibrinolytic system, which promotes the degradation of excessive extracellular matrix and scarring in the lungs. Experiments in mouse models and human cell lines demonstrated that this approach significantly reduces collagen deposition and restores fibrinolytic activity. This innovative strategy offers a potential transformative treatment for a progressive and often fatal respiratory disease.

References:

• Xu J, Xie Y, Luo Q, et al. Targeted DNA triplex-forming oligonucleotide liposome for pulmonary fibrosis gene therapy[J]. Cell Reports Medicine, 2026.