Selection of oligonucleotide aptamers with enhanced uptake and activation of human leukemia B cells

Hum Gene Ther. 2003 Jun 10;14(9):849-60. doi: 10.1089/104303403765701141.

Abstract

The clinical use of oligonucleotide (ODN) therapeutics has been hampered by their limited ability to penetrate intact cells. To identify ODN properties that would facilitate cellular uptake, we developed a repetitive selection procedure using an ODN library containing at least 10(14) different molecules and human B lymphoma cells as a target. Natural phosphodiester single-stranded DNA ODNs (R-aptamers) were obtained after 10 rounds of selection. A common feature in the R-aptamers was guanine-rich 3' terminal sequences, and many also contained potential immunostimulatory (ISS) CpG sequence motifs. Two R-aptamers (R10-60 and D-R15-8) with the predominant shared characteristics were selected for further study on primary human chronic lymphocytic leukemia (CLL) B cells, which are well known to be difficult to transfect and activate. Flow cytometry analysis of the CLL cells demonstrated that the fluorochrome-labeled R-aptamers were internalized much more efficiently than nonselected random sequence ODN. Studies on sequence modifications indicated that efficient uptake required ODN multimerization, that was promoted by guanine-rich sequences at the 3' terminus. In addition, CLL cells that were exposed to the aggregating R-aptamers containing CpG motifs were strongly activated, as indicated by upregulation of CD40 levels as compared to cells treated with nonaggregating CpG R-aptamers. Together, these findings suggest that the sequence compositions in R-aptamers that promote multimerization and contain optimal ISS CpG motifs facilitate the delivery of ISS-ODN to CLL cells and enhance the activation of these cells.

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adjuvants, Immunologic / chemistry*
  • Adjuvants, Immunologic / metabolism*
  • Adjuvants, Immunologic / pharmacology
  • Animals
  • Centrifugation, Density Gradient
  • Chromatography, High Pressure Liquid
  • DNA-Binding Proteins / immunology
  • DNA-Binding Proteins / metabolism
  • Gene Library
  • Humans
  • Leukemia, Lymphocytic, Chronic, B-Cell / immunology
  • Leukemia, Lymphocytic, Chronic, B-Cell / metabolism*
  • Mice
  • Oligodeoxyribonucleotides / chemistry
  • Oligodeoxyribonucleotides / metabolism
  • Oligodeoxyribonucleotides / pharmacology
  • Oligonucleotides / chemistry*
  • Oligonucleotides / metabolism*
  • Oligonucleotides / pharmacology
  • Permeability
  • Receptors, Cell Surface / analysis
  • Receptors, Cell Surface / immunology
  • Receptors, Cell Surface / metabolism
  • Reverse Transcriptase Polymerase Chain Reaction
  • Toll-Like Receptor 9
  • Tumor Cells, Cultured

Substances

  • Adjuvants, Immunologic
  • CPG-oligonucleotide
  • DNA-Binding Proteins
  • Oligodeoxyribonucleotides
  • Oligonucleotides
  • Receptors, Cell Surface
  • TLR9 protein, human
  • Tlr9 protein, mouse
  • Toll-Like Receptor 9