Central nervous system messenger RNA (CNS mRNA)

Central nervous system (CNS) messenger RNA (mRNA) sequences represent a broad class of therapeutic targets for RNA interference (RNAi) technologies, specifically small interfering RNAs (siRNAs). These targets are the intermediate transcripts that carry genetic information from DNA to the ribosome for protein synthesis; in the context of CNS diseases, these mRNAs often encode proteins that are misfolded, overexpressed, or toxic, such as huntingtin in Huntington's disease or tau in various dementias (Nature Reviews Drug Discovery, 2021). The therapeutic strategy involves delivering a synthetic siRNA that is perfectly complementary to a specific region of the target mRNA. Upon cellular uptake, the siRNA is incorporated into the RNA-induced silencing complex (RISC), which then uses the antisense strand to identify and cleave the target mRNA, thereby preventing the production of the disease-associated protein (Alnylam Pharmaceuticals, 2023). While this approach offers high specificity, the primary challenges include achieving effective delivery across the blood-brain barrier and ensuring that the silencing of the target gene does not interfere with essential biological processes or trigger an adverse immune response (PubMed, 2021).

Other names

CNS-expressed mRNABrain-expressed mRNANeuronal messenger RNATarget mRNA sequences

RNA interference (RNAi) mediated by the RNA-induced silencing complex (RISC), which facilitates the sequence-specific cleavage and degradation of target mRNA molecules.