title: Stable knockdown of microRNA in vivo by lentiviral vectors.
abstract: Studying microRNA function in vivo requires genetic strategies to generate loss-of-function phenotypes. We used lentiviral vectors to stably and specifically knock down microRNA by overexpressing microRNA target sequences from polymerase II promoters. These vectors effectively inhibited regulation of reporter constructs and natural microRNA targets. We used bone marrow reconstitution with hematopoietic stem cells stably overexpressing miR-223 target sequence to phenocopy the genetic miR-223 knockout mouse, indicating robust interference of microRNA function in vivo.
authors.affiliation: San Raffaele Telethon Institute for Gene Therapy, Via Olgettina 58, Milan, Italy.
authors.forename: Bernhard
authors.initials: B
authors.lastname: Gentner
authors.forename: Giulia
authors.initials: G
authors.lastname: Schira
authors.forename: Alice
authors.initials: A
authors.lastname: Giustacchini
authors.forename: Mario
authors.initials: M
authors.lastname: Amendola
authors.forename: Brian D
authors.initials: BD
authors.lastname: Brown
authors.forename: Maurilio
authors.initials: M
authors.lastname: Ponzoni
authors.forename: Luigi
authors.initials: L
authors.lastname: Naldini
mesh_terms: D000818:Animals; D001854:Bone Marrow Cells / Q000378:metabolism; D045744:Cell Line, Tumor; D005822:Genetic Vectors / Q000235:genetics*; D006412:Hematopoietic Stem Cells / Q000378:metabolism; D006801:Humans; D016086:Lentivirus / Q000235:genetics*; D051379:Mice; D018345:Mice, Knockout; D035683:MicroRNAs / Q000235:genetics*
chemical_list: D035683:MicroRNAs