Antibody-directed cell-type-specific delivery of siRNA.
Over the past four years, chemically synthesized short interfering RNA (siRNA) has become the standard tool for specific silencing of gene expression in vitro. The most difficult task in transferring this technology to an in vivo setting is to develop appropriate delivery strategies. With this aim, Song et al. recently reported the development of antibody-protamine fusion proteins as vehicles for receptor-directed delivery of siRNA. When a mixture of siRNA targeting tumor-related genes was administered in this way, tumor growth was inhibited in an engineered melanoma model, demonstrating the therapeutic potential of this technology. However, several challenges remain to be overcome before targeted gene silencing can become a reality for patients.
Vornlocher
Hans-Peter
HP
Alnylam Europe AG, Fritz-Hornschuch-Strasse 9, 95326 Kulmbach, Germany. hpvornlocher@alnylam.de
D000818:Animals; D000911:Antibodies, Monoclonal / Q000008:administration & dosage; D016503:Drug Delivery Systems; D020868:Gene Silencing; D006801:Humans; D007140:Immunoglobulin Fab Fragments / Q000008:administration & dosage; D066298:In Vitro Techniques; D009374:Neoplasms, Experimental / Q000235:genetics / Q000276:immunology / Q000628:therapy; D011479:Protamines / Q000008:administration & dosage; D034741:RNA, Small Interfering / Q000008:administration & dosage*; D011993:Recombinant Fusion Proteins / Q000008:administration & dosage
D000911:Antibodies, Monoclonal; D007140:Immunoglobulin Fab Fragments; D011479:Protamines; D034741:RNA, Small Interfering; D011993:Recombinant Fusion Proteins