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lüll Therapeutic regulation of gene expression in the inner ear using RNA interference Maeda Y; Sheffield AM; Smith RJHAdv Otorhinolaryngol 2009[]; 66 (ä): 13-36Targeting and downregulating specific genes with antisense and decoy oligonucleotides, ribozymes or RNA interference (RNAi) offer the theoretical potential of altering a disease phenotype. Here we review the molecular mechanism behind the in vivo application of RNAi-mediated gene silencing, focusing on its application to the inner ear. RNAi is a physiological phenomenon in which small, double-stranded RNA molecules (small interfering RNA, siRNA) reduce expression of homologous genes. Notable for its exquisite sequence specificity, it is ideally applied to diseases caused by a gain-of-function mechanism of action. Types of deafness in which gain-of-function mutations are observed include DFNA2 (KCNQ4), DFNA3 (GJB2) and DFNA5 (DFNA5). Several strategies can be used to deliver siRNA into the inner ear, including cationic liposomes, adeno-associated and lentiviral vectors, and adenoviral vectors. Transduction efficiency with cationic liposomes is low and the effect is transient; with adeno-associated and lentiviral vectors, long-term transfection is possible using a small hairpin RNA expression cassette.|*RNA Interference/physiology[MESH]|Animals[MESH]|Cochlear Aqueduct/physiology[MESH]|Connexin 26[MESH]|Connexins[MESH]|Gene Expression Regulation/physiology[MESH]|Gene Transfer Techniques[MESH]|Genetic Vectors[MESH]|Hearing Loss, Sensorineural/genetics[MESH]|Humans[MESH]|KCNQ Potassium Channels/genetics[MESH]|Labyrinth Diseases/*therapy[MESH]|Oligoribonucleotides, Antisense/physiology[MESH]|RNA, Catalytic/physiology[MESH]|Transfection[MESH] |