METHODS OF INHIBITING GENE EXPRESSION BY RNA INTERFERENCE
摘要
The invention provides a lentiviral vector capable of inhibiting the expression of at least one target gene. A lentiviral vector of the invention encompasses a first nucleic acid sequence derived from a target gene transcript and a second nucleic acid sequence corresponding to the reverse complement of said first nucleic acid sequence. A lentiviral vector of the invention capable of inhibiting the expression of a target gene is useful in therapeutic applications to inactivate disease-associated transcripts and thereby reduce the severity of inherited metabolic, infectious or malignant conditions. Methods for inhibiting one or more target genes in a cell as well as methods for producing a non-human mammal in which the expression of one or more target genes is inhibited also are provided by the present invention.
申请公布号
WO2004056966(A3)
申请公布日期
2004.09.02
申请号
WO2003US40948
申请日期
2003.12.18
申请人
SALK INSTITUTE FOR BIOLOGICAL STUDIES;VERMA, INDER, M.;TISCORNIA, GUSTAVO;SINGER, ODED