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LIPT2 antisense RNA 1 (LIPT2-AS1) is a long non-coding RNA transcribed antisense to the LIPT2 gene and predominantly expressed in human neural cells. It has been shown to produce a small open reading frame (smORF)-derived peptide, conserved across certain mammalian species, which contains a DNA-binding domain and may play a role in regulating gene expression during neuronal development. Its expression is high in the nervous system and is downregulated in certain brain tumors, such as large-cell medulloblastoma. Diseases loosely associated with mutations or altered expression include severe neonatal encephalopathy with lactic acidosis and brain abnormalities. Currently, there is no direct evidence for pharmacological targeting of LIPT2-AS1 or identification of interacting drugs, making it an RNA of biological interest rather than a canonical therapeutic target.
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