Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors

E Song, P Zhu, SK Lee, D Chowdhury… - Nature …, 2005 - nature.com
E Song, P Zhu, SK Lee, D Chowdhury, S Kussman, DM Dykxhoorn, Y Feng, D Palliser…
Nature biotechnology, 2005nature.com
Delivery of small interfering RNAs (siRNAs) into cells is a key obstacle to their therapeutic
application. We designed a protamine-antibody fusion protein to deliver siRNA to HIV-
infected or envelope-transfected cells. The fusion protein (F105-P) was designed with the
protamine coding sequence linked to the C terminus of the heavy chain Fab fragment of an
HIV-1 envelope antibody. siRNAs bound to F105-P induced silencing only in cells
expressing HIV-1 envelope. Additionally, siRNAs targeted against the HIV-1 capsid gene …
Abstract
Delivery of small interfering RNAs (siRNAs) into cells is a key obstacle to their therapeutic application. We designed a protamine-antibody fusion protein to deliver siRNA to HIV-infected or envelope-transfected cells. The fusion protein (F105-P) was designed with the protamine coding sequence linked to the C terminus of the heavy chain Fab fragment of an HIV-1 envelope antibody. siRNAs bound to F105-P induced silencing only in cells expressing HIV-1 envelope. Additionally, siRNAs targeted against the HIV-1 capsid gene gag, inhibited HIV replication in hard-to-transfect, HIV-infected primary T cells. Intratumoral or intravenous injection of F105-P-complexed siRNAs into mice targeted HIV envelope-expressing B16 melanoma cells, but not normal tissue or envelope-negative B16 cells; injection of F105-P with siRNAs targeting c-myc, MDM2 and VEGF inhibited envelope-expressing subcutaneous B16 tumors. Furthermore, an ErbB2 single-chain antibody fused with protamine delivered siRNAs specifically into ErbB2-expressing cancer cells. This study demonstrates the potential for systemic, cell-type specific, antibody-mediated siRNA delivery.
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