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449: siRNA divalente para doença priônica [PT]
28:46
Gentile JE et al., Nucleic Acids Research - Discovery and preclinical development of divalent siRNA candidates targeting PRNP, identifying 2439-s4 as a potent, durable human PRNP-lowering drug candidate with IND clearance. Key terms: prion disease, PrP lowering, divalent siRNA, 2439-s4, RNAi therapeutics.
Study Highlights: Authors screened divalent siRNA libraries and identified mouse-targeting…