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iPSC-Exo refers to exosomes derived from induced pluripotent stem cells (iPSCs). These nanoscale extracellular vesicles are secreted by iPSCs and can carry a variety of bioactive molecules, including proteins, mRNA, and microRNA. They have been investigated as therapeutic agents or drug delivery vehicles due to their ability to cross biological barriers such as the blood-brain barrier and modulate cellular processes in recipient cells. Mechanistically, iPSC-Exos exert effects through paracrine signaling—delivering their molecular cargo to target cells—which can regulate apoptosis, inflammation, fibrosis, angiogenesis, and immune responses. Preclinical studies have shown that loading drugs like amphotericin B into iPSC-Exos (forming an "iPSC-Exo/AmB" complex) enhances drug efficacy while reducing toxicity in models of central nervous system fungal infections[1]. Additionally, they are being explored for use in acute respiratory distress syndrome (ARDS)[5][8] and as cancer vaccines by leveraging shared antigens between tumor cells and iPSCs to stimulate anti-tumor immunity[6][7]. Current clinical development includes phase 2 trials for moderate-to-severe ARDS in children[5][8], with ongoing preclinical research into applications for CNS infections and cancer immunotherapy.
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