Small interfering RNA (siRNA) is a class of double-stranded RNA molecules, about 20–25 nucleotides long, that act as genetic “switches” inside the cell. Once introduced, siRNA specifically recognizes and silences messenger RNA (mRNA) that carries the instructions to produce a mutated or disease-causing protein. This natural cellular mechanism is called RNA interference (RNAi).

A simplified step-by-step process:

• The siRNA molecule is delivered into the cell

• It binds precisely to the target mRNA with complementary sequence

• The bound mRNA is degraded by the RNA-induced silencing complex (RISC)

• This prevents the production of the mutated protein

• Only the healthy allele remains active, allowing normal protein synthesis

• High specificity: targets only the mutated gene, sparing the healthy one

• Custom-designed: siRNA can be tailored to any known genetic mutation

• Broad applicability: from rare genetic diseases to metabolic, cardiovascular and oncological conditions

• Low off-target effects: advanced computational design minimizes unintended interactions