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Gapmer antisense oligonucleotide (ASO; 20-mer)

Linked experiment: EXP002000

Linked peptide: HIV-1 Tat CPP (recombinant Tat peptide)

Linked paper: Targeted Activation of Cystic Fibrosis Transmembrane Conductance Regulator (2019)

RNA fields

RNA Type

Gapmer antisense oligonucleotide (ASO; 20-mer)

RNA Payload Or Target

BGas (CFTR-AS1) lncRNA knockdown to activate CFTR transcription (gapmer 10, G10; targets BGas intron 1)

RNA Modifications

20-nt gapmer with phosphorothioate (PS) bonds: 3 PS at both 5′ and 3′ ends surrounding an unmodified DNA-like gap; scramble control also PS-modified.