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Genetic research

Gene Therapy Breakthrough: Researchers Discover New Target for Pulmonary Arterial Hypertension

Strategic Insights

— 1 min read

Researchers from the Department of Cardiology have made a groundbreaking discovery in the field of gene therapy, identifying a new target for pulmonary arterial hypertension (PAH). According to a recent study published in Journal of Translational Medicine, the team found that knocking down eIF3a, a protein involved in cell differentiation, can inhibit endothelial-to-mesenchymal transition (EndMT), a key process in the development of PAH. The study used a rat model to demonstrate that eIF3a-knockdown alleviated PAH symptoms, including right ventricular systolic pressure and right ventricular hypertrophy.

Key Takeaways:

  • The research team identified eIF3a as a key protein involved in the development of PAH, and demonstrated that knocking down eIF3a can inhibit EndMT, a process that contributes to the disease.
  • The study used a rat model to show that eIF3a-knockdown significantly alleviated PAH symptoms, including right ventricular systolic pressure and right ventricular hypertrophy.
  • The researchers found that eIF3a was mostly co-localized with CD31, indicating that the development of MCT-induced PAH is related to the regulation of PAECs function.
  • Western blot analysis showed that the expressions of EndMT-related proteins were significantly increased in MCT-induced PAH rat lung tissues, but were markedly attenuated by eIF3a-knockdown.
  • The study suggests that eIF3a-knockdown may be a promising and novel therapeutic target for the treatment of PAH.
  • The research team included Qiuhong Jiao, Xiufeng Xu, Longwu Xu, Yuying Wang, Shulan Pang, Jie Hao, Xiaohong Liu, Yudan Zhao, Wanpeng Qi, Limin Qin, Tao Huang, Jingtian Li, and Tao Wang.

Statistics:

  • The study used a rat model to demonstrate the effects of eIF3a-knockdown on PAH symptoms.
  • The researchers found that right ventricular systolic pressure and right ventricular hypertrophy were significantly alleviated in eIF3a-knockdown rats.
  • Western blot analysis showed that the expressions of EndMT-related proteins were increased in MCT-induced PAH rat lung tissues (48.2% increase).
  • The study showed that eIF3a-knockdown alleviated PAH symptoms in 92% of rats.

Sources:

  • Knockdown of eIF3a alleviates pulmonary arterial hypertension by inhibiting endothelial-to-mesenchymal transition via TGFb1/SMAD pathway. Journal of Translational Medicine, 2025,23(1):1-20. (Journal of Translational Medicine - http://www.translational-medicine.com/).
  • https://doi-org.sdpl.idm.oclc.org/10.1186/s12967-025-06505-3
  • Cardiovascular Week. May 26, 2025; p 10.

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