ASYMMETRIC SHORT DUPLEX DNA AS A NOVEL GENE SILENCING TECHNOLOGY AND USE THEREOF

The present invention discloses a novel type of gene silencing technology for modulation of target nucleic acid and/or protein in cells, tissues, organisms and animals. The new technology provides compositions for use in gene targeting or gene silencing applications, including prevention and treatme...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: SUN, Xiangao, LI, Charles, LI, Chiang
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung:The present invention discloses a novel type of gene silencing technology for modulation of target nucleic acid and/or protein in cells, tissues, organisms and animals. The new technology provides compositions for use in gene targeting or gene silencing applications, including prevention and treatment of human diseases. The composition comprises an asymmetric, short, duplex DNA molecule where the sense strand is shorter than the antisense strand. The duplex DNA molecule further includes at least one interspersed segment of ribonucleotide monomer. The present invention further provides methods of using the compositions for modulating expression or function of a target gene, or for treatment or prevention of diseases as well as for other medical or biological applications.