The present application belongs to the technical field of
biomedicine, and provides a pharmaceutical composition for inhibiting NFKBIZ
gene expression, and a use. The present application designs a double-stranded
nucleic acid molecule capable of targeting and regulating NFKBIZ
gene expression. By means of verifying the
gene regulation efficiency of
nucleic acid molecules, multiple
nucleic acid molecules capable of inhibiting NFKBIZ
gene expression are obtained from screening, and efficient inhibition of IκB-ζ
protein expression is achieved at the
protein level. In addition, the present application verifies a use of a pharmaceutical composition formed by the conjugation of the described nucleic acid molecules and targeting ligand molecules in ophthalmic diseases. The pharmaceutical composition can solve the problems with existing
small molecule immunomodulatory drugs of low
delivery efficiency and poor
therapeutic effect, providing small nucleic acid drugs having tissue-targeted delivery functions, and has the advantages of long duration of
efficacy and low side effects. In addition, the pharmaceutical composition has simple ingredients, is easy to synthesize, and has good prospects for translational applications.