Dual functional BAFF receptor aptamers inhibit ligand-induced proliferation and deliver siRNAs to NHL cells

Nucleic Acids Res. 2013 Apr;41(7):4266-83. doi: 10.1093/nar/gkt125. Epub 2013 Mar 6.

Abstract

The B-cell-activating factor (BAFF)-receptor (BAFF-R) is restrictedly expressed on B-cells and is often overexpressed in B-cell malignancies, such as non-Hodgkin's lymphoma. On binding to its ligand BAFF, proliferation and cell survival are increased, enabling cancer cells to proliferate faster than normal B-cells. Nucleic acid aptamers can bind to target ligands with high specificity and affinity and may offer therapeutic advantages over antibody-based approaches. In this study, we isolated several 2'-F-modified RNA aptamers targeting the B-cell-specific BAFF-R with nanomolar affinity using in vitro SELEX technology. The aptamers efficiently bound to BAFF-R on the surface of B-cells, blocked BAFF-mediated B-cell proliferation and were internalized into B-cells. Furthermore, chimeric molecules between the BAFF-R aptamer and small interfering RNAs (siRNAs) were specifically delivered to BAFF-R expressing cells with a similar efficiency as the aptamer alone. We demonstrate that a signal transducer and activator of transcription 3 (STAT3) siRNA delivered by the BAFF-R aptamer was processed by Dicer and efficiently reduced levels of target mRNA and protein in Jeko-1 and Z138 human B-cell lines. Collectively, our results demonstrate that the dual-functional BAFF-R aptamer-siRNA conjugates are able to deliver siRNAs and block ligand mediated processes, suggesting it might be a promising combinatorial therapeutic agent for B-cell malignancies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aptamers, Nucleotide / chemistry
  • Aptamers, Nucleotide / metabolism
  • Aptamers, Nucleotide / pharmacology*
  • B-Cell Activating Factor / antagonists & inhibitors*
  • B-Cell Activation Factor Receptor / metabolism*
  • Cell Line
  • Cell Proliferation
  • Humans
  • L-Selectin / genetics
  • L-Selectin / metabolism
  • Ligands
  • RNA Interference
  • RNA, Small Interfering / administration & dosage*
  • STAT3 Transcription Factor / genetics
  • STAT3 Transcription Factor / metabolism

Substances

  • Aptamers, Nucleotide
  • B-Cell Activating Factor
  • B-Cell Activation Factor Receptor
  • Ligands
  • RNA, Small Interfering
  • STAT3 Transcription Factor
  • TNFRSF13C protein, human
  • L-Selectin