title: Molecular umbrella conjugate for the ocular delivery of siRNA.
abstract: The synthesis, computer modeling, and biological activity of an octawalled molecular umbrella short interfacing RNA (siRNA) conjugate is described. This molecular umbrella-siRNA conjugate exhibited mRNA knockdown activity in vitro in the absence of a transfection reagent. Evaluation of this molecular umbrella conjugate in vivo, using the rat eye via intravitreal injection, resulted in sequence specific mRNA knockdown in the retina with no obvious signs of toxicity, as judged by ophthalmic examination.
authors.affiliation: Department of Chemistry, Lehigh University , Bethlehem, Pennsylvania 18015, United States.
authors.forename: Vaclav
authors.initials: V
authors.lastname: Janout
authors.forename: Lauren L
authors.initials: LL
authors.lastname: Cline
authors.forename: Bradley P
authors.initials: BP
authors.lastname: Feuston
authors.forename: Lee
authors.initials: L
authors.lastname: Klein
authors.forename: Ann
authors.initials: A
authors.lastname: O'Brien
authors.forename: Tom
authors.initials: T
authors.lastname: Tucker
authors.forename: Yu
authors.initials: Y
authors.lastname: Yuan
authors.forename: Lynn A
authors.initials: LA
authors.lastname: O'Neill-Davis
authors.forename: Robert L
authors.initials: RL
authors.lastname: Peiffer
authors.forename: Sandhya S
authors.initials: SS
authors.lastname: Nerurkar
authors.forename: Vasant
authors.initials: V
authors.lastname: Jadhav
authors.forename: David M
authors.initials: DM
authors.lastname: Tellers
authors.forename: Steven L
authors.initials: SL
authors.lastname: Regen
mesh_terms: D004333:Drug Administration Routes; D004337:Drug Carriers; D005123:Eye; D057809:HEK293 Cells; D006801:Humans; D056004:Molecular Dynamics Simulation; D034741:RNA, Small Interfering / Q000008:administration & dosage*
chemical_list: D004337:Drug Carriers; D034741:RNA, Small Interfering