Muhammad Moazzam, Mengjie Zhang, Abid Hussain, Xiaotong Yu, Jia Huang, Yuanyu Huang
Five siRNA-based therapeutics have been approved by the Food and Drug Administration (FDA), including patisiran, givosiran, lumasiran, inclisiran and vutrisiran. Besides, siRNA delivery to the target site without toxicity is a big challenge for researchers, and naked-siRNA delivery possesses several challenges, including membrane impermeability, enzymatic degradation, mononuclear phagocyte system (MPS) entrapment, fast renal excretion, endosomal escape, and off-target effects. The siRNA therapeutics can silence any disease-specific gene, but their intracellular and extracellular barriers limit their clinical applications...
January 9, 2024: Molecular Therapy