Innovative Gene Therapy Developed by Biopharmaceutical Research Institute-Led Team Offers Promising Treatment for Acute Lung Injury

Translated by Chance Lai
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A groundbreaking study led by a collaborative team of researchers from the Institute of Biopharmaceutical Sciences at National Yang Ming Chiao Tung University (NYCU), including Professors Chi-Ying Huang and Ly James Lee, Dr. Hon-Kan Yip from Kaohsiung Chang Gung Memorial Hospital, Deputy Director Chih-Hung Chang from Far Eastern Memorial Hospital, and Researcher Hsiu-Jung Liao, has unveiled a novel gene therapy utilizing extracellular vesicles (EVs) encapsulating nucleic acid drugs.

This innovation shows potential in combating severe respiratory diseases such as Acute Lung Injury (ALI), demonstrating modified EVs' significant ability to reduce lung inflammation and fibrosis. The research findings have been published in the prestigious 'Journal of Biomedical Science'.

Addressing COVID-19 Complications: Pioneering Research in Acute Respiratory Distress Syndrome (ARDS)

According to the team, in the wake of the global COVID-19 pandemic, the severe rate of COVID-19 cases stands at approximately 5%, often accompanied by Acute Respiratory Distress Syndrome (ARDS). However, despite significant advancements in respiratory medical technology, studies have shown that the mortality rate for severe ARDS remains as high as 88%.

Professor Chi-Ying Huang explained that the inspiration for this research stemmed from Dr. Hon-Kan Yip's clinical study on umbilical cord mesenchymal stem cell therapy. Through an extensive literature review, they found numerous teams exploring the application of stem cells and their derived extracellular vesicles in treating acute lung injuries.

However, the research team believed extracellular vesicles derived from stem cells had limited effectiveness. Therefore, they actively sought feasible target genes. Given previous studies showing the efficacy of traditional Chinese medicine in alleviating COVID symptoms, mainly linked to upregulating intracellular "let7a-5p", a molecule crucial in regulating inflammatory factors, the team decided to target let7a-5p as the key therapeutic gene.

Subsequently, leveraging Professor Ly James Lee's developed cell electroporation transfection technique, umbilical cord mesenchymal stem cells were engineered to produce extracellular vesicles rich in "let7a-5p" microRNA, offering a novel approach for treating lung injuries.

The team from NYCU then took charge of manufacturing and purifying extracellular vesicle nucleic acid drugs, followed by immunological tests conducted in collaboration with Far Eastern Memorial Hospital and animal studies validation with the team from Kaohsiung Chang Gung Memorial Hospital.

Advancing Biopharmaceutical Innovation: Engineered Extracellular Vesicles Offer New Hope in Treating Acute Lung Injury

The research results indicate that this therapy outperforms stem cell-derived extracellular vesicles, exhibiting superior immunomodulatory and anti-fibrotic effects. The significant research achievement, titled "Engineered extracellular vesicles carrying let-7a-5p for alleviating inflammation in acute lung injury," was published in the Journal of Biomedical Science.

The research team highlighted that extracellular vesicle nucleic acid drugs represent a novel biopharmaceutical, with current international research focusing on their modification, large-scale purification, and production processes. They anticipate that they can introduce different concepts to drug development through their unique techniques.

The research team is developing next-generation cell electroporation chip technology for large-scale manufacturing and commercialization. They also plan to advance extracellular vesicle nucleic acid drugs towards clinical applications in the future.