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TUBB2A mRNA encodes the Tubulin beta-2A chain, a critical structural component of microtubules primarily expressed in the developing central nervous system (2, 6). Microtubules are essential for various cellular processes, including mitosis, intracellular transport, and neuronal migration (3, 7). Mutations in the TUBB2A gene are associated with a spectrum of neurodevelopmental disorders known as tubulinopathies, characterized by cortical malformations and epilepsy (4, 9). Additionally, TUBB2A has been identified as a potential therapeutic target in certain cancers, such as triple-negative breast cancer and T-cell acute lymphoblastic leukemia, where its overexpression is linked to poor prognosis and immune evasion (1, 22). While traditional tubulin-targeting drugs act on the protein level, TUBB2A mRNA is an emerging target for precision medicine approaches such as antisense oligonucleotides (ASOs) or RNA interference (RNAi) to address gain-of-function mutations or pathological overexpression (17, 19).
RNA interference (RNAi) or antisense-mediated degradation to reduce protein expression levels.
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