Myostatin pre-mRNA (MSTN pre-mRNA)

Myostatin pre-mRNA" refers specifically to the unspliced precursor mRNA transcribed from the MSTN gene, which encodes myostatin, also known as growth differentiation factor 8 (GDF-8). Myostatin pre-mRNA undergoes splicing and processing to generate the mature myostatin mRNA, which is then translated into the myostatin pro-protein and later processed into its active form. While myostatin protein is a well-established negative regulator of skeletal muscle growth and a target in muscle-wasting conditions, "myostatin pre-mRNA" as a target is highly specific and not a canonical protein, enzyme, or receptor target. Antisense oligonucleotides (such as KMM001) have been developed to selectively inhibit maturation of myostatin pre-mRNA, blocking myostatin production at the transcriptional level. This strategy is under investigation for potential therapeutic benefit in muscle-wasting diseases, but the biological and clinical roles of targeting the pre-mRNA are still emerging. *Note:* Most conventional drug targets focus on the mature protein rather than its precursor mRNA, so this target designation is atypical and may not align with standard classification systems for therapeutic targets.

Other names

Myostatin transcriptMSTN pre-mRNAGrowth differentiation factor 8 pre-mRNA

Inhibition of pre-mRNA splicing to reduce mature myostatin protein levels