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Brain-derived neurotrophic factor (BDNF) mRNA is an mRNA-based biologic therapeutic designed to treat ischemic neuronal death, such as that occurring after a stroke or transient global ischemia. The therapeutic consists of synthetic mRNA encoding the BDNF protein, encapsulated within a polyplex nanomicelle—a polymer-based carrier designed to protect the mRNA from degradation and facilitate its delivery into target cells. Upon administration, typically via the intraventricular route in preclinical models, the mRNA is translated into BDNF protein within the brain. BDNF then acts as a potent neurotrophic factor, promoting the survival, growth, and differentiation of neurons by activating the Tropomyosin receptor kinase B (TrkB) signaling pathway. Preclinical studies in rat models have demonstrated that this approach can significantly increase the survival of hippocampal neurons and improve spatial memory, particularly when administered in multiple doses following the ischemic event.
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