IncRNA FENDRR Exhibits Anti-fibrotic Activity in Pulmonary Fibrosis

Case ID:
2020-010

BACKGROUND

Idiopathic pulmonary fibrosis (IPF) is a fatal fibrotic lung disease characterized by a progressive decline in lung function that is chronic and fatal. The only treatment option shown to significantly extend life in a patient with IPF is a lung transplant, which only provides a ~40% survival rate 5 years post-operation. IPF usually occurs in people between the ages of 50 and 70, affecting approximately 200,000 individuals and causing 40,000 annually in America each year. Unfortunately, limited knowledge of pathogenesis of IPF has resulted in a lack of effective treatments to halt, or reverse IPF. Among non-coding RNAs, microRNAs have been extensively studied in IPF. However, little is known regarding the roles of lncRNAs in IPF. Fetal-lethal noncoding developmental regulatory RNA (FENDRR) is a lncRNA that is transcribed bidirectionally with FOXF1 on its opposite strand. FENDRR binds to polycomb repressive complex 2 (PRC2) and/or TrxG/MLL complexes to epigenetically regulate the expression of its target genes.  FENDRR is known to be essential for lung development. FENDRR had the fifth and third highest expression among all of the lncRNAs in normal human lungs and human pulmonary fibroblasts.

SUMMARY OF TECHNOLOGY

OSU investigators have confirmed down-regulation of FENDRR in the lungs using real-time PCR in an independent cohort of 27 patients with IPF from the Lung Tissue Research Consortium. They discovered that FENDRR is down-regulated in fibrotic fibroblasts via TGFβ1-SMAD3 signaling. Their results further demonstrated that FENDRR reduced pulmonary fibrosis by binding iron-responsive element-binding protein 1 (IRP1) to control iron levels and by competing with the pro-fibrotic miR-214.

 

Adenovirus-mediated FENDRR overexpression in the mouse lung reduced collagen content, attenuated bleomycin-induced lung fibrosis and improved lung function.

 

Figure 1 The down-regulation of FENDRR in the lungs was confirmed using real-time PCR in an independent cohort of 27 patients with IPF from the Lung Tissue Research Consortium (LTRC) and 6 human adult normal lung tissues from BioChain.

 

 

 

 

Figure 2  (A) Shows a 1.7-fold and 3.5-fold increases in FENDRR expression in the lungs of saline control- and bleomycin-treated mice, respectively; increased FENDRR expression has led to (B) reduced bleomycin-induced COL1A1 mRNA expression and (C), (D) protein expression of COL1A1.

POTENTIAL AREAS OF APPLICATION

  • FENDRR is a potential therapeutic target for IPF
  • Treatment of IPF using FENDRR gene therapy

STAGE OF DEVELOPMENT

  • Proof-of-concept

 

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Patent Information:
For Information, Contact:
Russell Hopper
Sr. Licensing Associate
Oklahoma State University
russell.hopper@okstate.edu
Inventors:
Chaoqun Huang
Lin Liu
Keywords:
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