CFTR antisense RNA 1 (CFTR-AS1)

CFTR antisense RNA 1 (CFTR-AS1, also known as BGas) is a long non-coding RNA (lncRNA) transcribed in the antisense orientation from within intron 11 of the CFTR gene. BGas/CFTR-AS1 is approximately 3,944 nucleotides in length and is localized in the nucleus. It interacts with chromatin remodeling proteins (such as HMGA1, HMGB1, and WIBG) at the CFTR gene locus, where it modulates local chromatin structure and inhibits CFTR gene transcription by disrupting RNA polymerase II function. This results in decreased CFTR mRNA and protein levels, ultimately reducing CFTR chloride channel activity. Targeting CFTR-AS1 with antisense oligonucleotides (ASOs) can downregulate this lncRNA, enhance CFTR transcription, and restore protein function, suggesting potential as a therapeutic strategy for cystic fibrosis by increasing endogenous CFTR expression[2].

Other names

BGasB GasCFTR-AS1

Antisense oligonucleotide binding to CFTR-AS1, leading to RNase H-mediated degradation of the RNA, which enhances CFTR gene transcription and protein expression by removing the inhibitory effect of CFTR-AS1 on the CFTR gene