title: Allele-specific silencing by RNA interference.
abstract: Allele-specific gene silencing by RNA interference (RNAi) is therapeutically useful for specifically inhibiting the expression of disease-associated alleles without suppressing the expression of corresponding wild-type alleles. To realize such allele-specific RNAi (ASP-RNAi), the design and assessment of small interfering RNA (siRNA) duplexes conferring ASP-RNAi is vital; however, it is also difficult. We have developed an assay system with reporter alleles that encode the Photinus and Renilla luciferase genes carrying mutant and wild-type allelic sequences in their 3'-untranslated regions. The assay system allows us to evaluate designed siRNAs and also short hairpin RNAs for allele-specific silencing against target mutant alleles as well as off-target silencing against corresponding wild-type alleles simultaneously in a qualitative and quantitative manner.
authors.affiliation: National Institute of Neuroscience, NCNP, Tokyo, Japan.
authors.forename: Hirohiko
authors.initials: H
authors.lastname: Hohjoh
mesh_terms: D000483:Alleles; D000818:Animals; D005821:Genetic Techniques; D006801:Humans; D049409:Luciferases, Firefly / Q000235:genetics; D049410:Luciferases, Renilla / Q000235:genetics; D034622:RNA Interference; D034741:RNA, Small Interfering
chemical_list: D034741:RNA, Small Interfering; C485197:luciferase, Photinus; D049410:Luciferases, Renilla; D049409:Luciferases, Firefly