CFTR antisense RNA 2 (CFTR-AS2)

CFTR antisense RNA 2 (CFTR-AS2) is a long non-coding RNA transcribed from the CFTR gene locus, running antisense to the coding direction of CFTR. It is thought to play a regulatory role in CFTR gene expression, potentially affecting mRNA processing, stability, and degradation. CFTR-AS2 is being studied for its relevance in cystic fibrosis pathology, particularly as an RNA-based target for therapies aiming to restore or modulate CFTR protein production in patients with nonsense or splicing mutations. Although direct therapeutic targeting of CFTR-AS2 has not yet reached the clinic, its regulatory functions may inform future RNA-based therapies for cystic fibrosis.

Other names

CFTR-AS2CFTR Antisense RNA 2

Not applicable for drugs directly targeting CFTR-AS2; ASOs modulate splicing, stability, or translation of CFTR mRNA, potentially indirectly involving CFTR-AS2