Lysine methyltransferase 2A (KMT2A) mRNA (KMT2A mRNA)

KMT2A (Lysine methyltransferase 2A) mRNA is the transcript of the KMT2A gene, which encodes a critical histone methyltransferase responsible for methylating histone H3 at lysine 4 (H3K4). This epigenetic modification is essential for maintaining the expression of developmental genes, particularly the HOX cluster, which governs hematopoiesis and embryonic patterning. In many aggressive acute leukemias, the KMT2A gene undergoes chromosomal translocations, resulting in chimeric mRNAs that produce oncogenic fusion proteins. Targeting the KMT2A mRNA directly via RNA-based therapeutics, such as small interfering RNAs (siRNAs) or antisense oligonucleotides (ASOs), represents a strategy to silence the expression of these drivers at the pre-translational level. While clinical development has largely focused on small-molecule inhibitors of the KMT2A-Menin protein interaction, mRNA-directed approaches are being explored to address both leukemias and solid tumors where KMT2A is overexpressed. Additionally, KMT2A mRNA levels are a key focus in understanding the pathogenesis of Wiedemann-Steiner syndrome, a neurodevelopmental disorder caused by haploinsufficiency of the gene.

Other names

MLL mRNAMLL1 mRNAHRX mRNAALL-1 mRNACXXC7 mRNAHTRX1 mRNATRX1 mRNAMixed lineage leukemia mRNA

RNA interference (siRNA) and antisense-mediated mRNA degradation (ASO) leading to reduced translation of the KMT2A protein or its oncogenic fusion variants.