Alpha-1 antitrypsin mRNA (A1AT mRNA)

Alpha-1 antitrypsin mRNA comprises a set of transcripts produced by the *SERPINA1* gene, primarily in liver and lung cells. These mRNAs feature diverse 5'-untranslated regions due to alternative splicing, which influence translation efficiency and tissue-specific expression. The resulting protein, alpha-1 antitrypsin, is a major serine protease inhibitor that protects tissues from proteolytic damage, especially in the lungs. Inherited mutations affecting SERPINA1 mRNA structure or sequence can lead to insufficient protein production, resulting in alpha-1 antitrypsin deficiency, which manifests as liver and lung disease. Experimental therapies delivering functional SERPINA1 mRNA aim to restore circulating alpha-1 antitrypsin and ameliorate disease. In summary: Alpha-1 antitrypsin mRNA is relevant as a target for gene and RNA therapies, but is not a classical pharmacological target. Standardized target information (e.g., for a database) should prefer the protein "Alpha-1 antitrypsin" as canonical. For RNA or gene therapy contexts, Alpha-1 antitrypsin mRNA is a valid target entry, with the caveat noted above.

Other names

Alpha-1 antitrypsin transcriptSERPINA1 mRNAA1AT mRNAα1-antitrypsin mRNA

mRNA replacement therapy: Delivery of synthetic SERPINA1 mRNA to liver cells produces functional alpha-1 antitrypsin protein, compensating for genetic deficiency