New stem cell model can help personalize stem cell treatment for immunodeficiency patients

Graphical abstract. Credit: Journal of Allergy and Clinical Immunology (2023). DOI: 10.1016/j.jaci.2023.11.914

A collaborative research team has pioneered a new stem cell model to help personalize treatment for patients suffering from rare forms of immunodeficiency. The research findings were published in the Journal of Allergy and Clinical Immunology.

Primary immunodeficiencies, also known as “inborn errors of immunity,” are debilitating diseases that compromise the immune system, leaving patients highly vulnerable to infections, autoimmunity, and even cancer. To date, about 500 primary immunodeficiencies are known, but the list is growing yearly as new diseases emerge. One example is a rare disorder called STAT1-Gain-of-Function (STAT1-GoF) disease. Patients with STAT1-GoF are born with an inheritable defect in their immune system, making them susceptible to life-threatening infections, autoimmune disorders, aneurysms, and cancers.

Collaborating with partners at the Centre for Translational Stem Cell Biology (CTSCB) and the University of Cambridge, HKUMed has pioneered a new stem cell platform to help patients with primary immunodeficiencies. The research team led by Dr. Philip Li Hei, Professors Liu Pengtao, and Chak-sing Lau from the LKS Faculty of Medicine of the University of Hong Kong (HKUMed) took blood samples from patients and re-engineered the patients’ cells into Expanded Potential Stem Cells (EPSCs), which can be used as personalized disease models, enabling various therapies to be tested to identify the most effective and safest treatment options without causing unnecessary risk to the patients.

By The University of Hong Kong

Article can be accessed on: MedicalXpress