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Nucleoporin 205 (NUP205) mRNA is the transcript encoding a critical scaffold protein of the nuclear pore complex (NPC) inner ring. The NPC is the essential gateway for the bidirectional transport of proteins and RNA between the nucleus and cytoplasm, maintaining cellular homeostasis [1, 2]. Mutations in the NUP205 gene are a known cause of steroid-resistant nephrotic syndrome (SRNS), where disrupted NPC function leads to podocyte injury and renal failure [3]. Additionally, NUP205 mRNA is frequently overexpressed in various cancers, such as hepatocellular carcinoma and glioblastoma, where it promotes tumor cell proliferation and migration [4, 5]. While no drugs are currently approved for this target, NUP205 mRNA is being investigated as a target for RNA-based therapies like siRNA and antisense oligonucleotides to modulate its expression in disease states [5, 6]. However, because the NPC is fundamental to all eukaryotic cells, therapeutic targeting of NUP205 mRNA carries a high risk of systemic toxicity due to the potential for widespread impairment of cellular transport [2].
Antisense inhibition; RNA interference
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