Quantification of cytosolic plasmid DNA degradation using high-throughput sequencing: implications for gene delivery

J Gene Med. 2014 Mar-Apr;16(3-4):75-83. doi: 10.1002/jgm.2761.

Abstract

Background: Although cytosolic DNA degradation plays an important role in decreasing transgene expression, the plasmid degradation pattern remains largely unexplored.

Methods: Illumina dye sequencing was employed to provide degradation site information for S1 and cytosolic nucleases. S1 nuclease provided a positive control for a comparison between the agarose gel method and sequencing approaches.

Results: The poly(A) region between the β-lactamase gene and the cytomegalovirus (CMV) promoter was identified as the most likely cut site for polyplex-treated cytosol. The second most likely site, at the 5' end of the β-lactamase gene, was identified by gel electrophoresis and sequencing. Additional sites were detected in the OriC region, the SV40/poly(A) region, the luciferase gene and the CMV promoter. Sequence analysis of plasmid treated with cytosol from control cells showed the greatest cut activity in the OriC region, the β-lactamase gene and the poly(A) region following the luciferase gene. Additional regions of cut activity include the SV40 promoter and the β-lactamase poly(A) termination sequence. Both cytosolic nucleases and the S1 nuclease showed substantial activity at the bacterial origin of replication (OriC).

Conclusions: High-throughput plasmid sequencing revealed regions of the luciferase plasmid DNA sequence that are sensitive to cytosolic nuclease degradation. This provides new targets for improving plasmid and/or polymer design to optimize the likelihood of protein expression.

Keywords: HeLa; biotechnology; plasmid design; plasmid gene delivery; polymer vector.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Cytomegalovirus / genetics
  • DNA / metabolism*
  • Electrophoresis, Agar Gel
  • Endonucleases / genetics
  • Endonucleases / metabolism
  • Gene Transfer Techniques*
  • Genetic Therapy
  • Genetic Vectors
  • High-Throughput Nucleotide Sequencing / methods*
  • Luciferases / genetics
  • Luciferases / metabolism
  • Origin Recognition Complex / genetics
  • Plasmids / metabolism*
  • Poly(A)-Binding Proteins / genetics
  • Poly(A)-Binding Proteins / metabolism
  • Promoter Regions, Genetic
  • Sequence Analysis, DNA
  • Transgenes
  • beta-Lactamases / genetics

Substances

  • OriC chromosomal replication origin
  • Origin Recognition Complex
  • Poly(A)-Binding Proteins
  • DNA
  • Luciferases
  • Endonucleases
  • beta-Lactamases