title: Manipulating the in vivo immune response by targeted gene knockdown.
abstract: Aptamers, nucleic acids selected for high affinity binding to proteins, can be used to activate or antagonize immune mediators or receptors in a location and cell-type specific manner and to enhance antigen presentation. They can also be linked to other molecules (other aptamers, siRNAs or miRNAs, proteins, toxins) to produce multifunctional compounds for targeted immune modulation in vivo. Aptamer-siRNA chimeras (AsiCs) that induce efficient cell-specific knockdown in immune cells in vitro and in vivo can be used as an immunological research tool or potentially as an immunomodulating therapeutic.
authors.affiliation: Program in Cellular and Molecular Medicine, Boston Children's Hospital and Department of Pediatrics, Harvard Medical School, 200 Longwood Avenue, Boston, MA 02115, USA. Electronic address: judy.lieberman@childrens.harvard.edu.
authors.forename: Judy
authors.initials: J
authors.lastname: Lieberman
mesh_terms: D000818:Animals; D052157:Aptamers, Nucleotide / Q000138:chemical synthesis / Q000627:therapeutic use*; D055785:Gene Knockdown Techniques; D006801:Humans; D056747:Immunomodulation; D007167:Immunotherapy / Q000379:methods*; D058990:Molecular Targeted Therapy; D034741:RNA, Small Interfering / Q000138:chemical synthesis / Q000627:therapeutic use*
chemical_list: D052157:Aptamers, Nucleotide; D034741:RNA, Small Interfering