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USH2A antisense RNA 1 (USH2A-AS1) is a long non-coding RNA (lncRNA) that is transcribed antisense to the USH2A gene region. There is currently no direct evidence that USH2A-AS1 itself serves as a therapeutic target, disease biomarker, or has established biological function or involvement in disease pathways. Its biological role is undetermined; in general, antisense RNAs can participate in the regulation of gene expression for their sense gene partners, but such a function has not been demonstrated for USH2A-AS1. Most literature and disease relevance pertain to the USH2A gene (usherin), not to its antisense transcript USH2A-AS1[2][4]. Key clarifications: - USH2A-AS1 is **not** the same as the USH2A gene (which encodes the usherin protein, a major disease gene in Usher syndrome type 2A and some forms of retinitis pigmentosa)[1][5][6][7]. - Almost all clinical research and therapeutic activity (e.g., antisense oligonucleotides such as QR-421a) target the *USH2A* **gene** (and its mRNA/protein product), not the antisense transcript USH2A-AS1[7][9]. - There is no evidence that USH2A-AS1 is directly targeted by drugs or considered a therapeutic target of current interest. If you are seeking information on the *USH2A* gene (usherin), clarify your intent and use the correct target name to avoid confusion.
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