E- vectors: development of novel self-inactivating and self-activating retroviral vectors for safer gene therapy

J Virol. 1995 Nov;69(11):6839-46. doi: 10.1128/JVI.69.11.6839-6846.1995.

Abstract

We have developed novel self-inactivating and self-activating retroviral vectors based on the previously observed high-frequency deletion of direct repeats. We constructed spleen necrosis virus (SNV)-based viral vectors that contained large direct repeats flanking the viral encapsidation sequence (E). A large proportion of the proviruses in the target cells had E and one copy of the direct repeat deleted. Direct repeats of 1,333 and 788 bp were deleted at frequencies of 93 and 85%, respectively. To achieve a 100% deletion efficiency in target cells after ex vivo infection and drug selection, we constructed a self-activating vector that simultaneously deleted E and reconstituted the neomycin phosphotransferase gene. Selection of the target cells for resistance to G418 (a neomycin analog) ensured that all integrated proviruses had E deleted. The proviruses with E deleted were mobilized by a replication-competent virus 267,000-fold less efficiently than proviruses with E. We named these self-inactivating vectors E- (E-minus) vectors. These vectors should increase the safety of retroviral vector-mediated gene therapy by preventing the spread of vector sequences to nontarget cells in the event of coinfection with helper virus. We propose that direct-repeat deletions occur during RNA-dependent DNA synthesis and suggest that template switches occur without a requirement for RNA breaks. The minimum template dissociation frequency was estimated as 8%/100 bp per replication cycle. These vectors demonstrate that large direct repeats and template-switching properties of reverse transcriptase can be utilized to delete any sequence or reconstitute genes during retroviral replication.

Publication types

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

MeSH terms

  • Animals
  • Cell Line
  • Cloning, Molecular
  • Genetic Therapy / methods*
  • Genetic Therapy / standards
  • Genetic Vectors*
  • Humans
  • Kanamycin Kinase
  • Phosphotransferases (Alcohol Group Acceptor) / biosynthesis
  • Proviruses / genetics
  • RNA, Viral / biosynthesis
  • Recombinant Proteins / biosynthesis
  • Repetitive Sequences, Nucleic Acid*
  • Restriction Mapping
  • Retroviridae* / genetics
  • Safety
  • Sequence Deletion*
  • Transfection
  • Virus Integration

Substances

  • RNA, Viral
  • Recombinant Proteins
  • Phosphotransferases (Alcohol Group Acceptor)
  • Kanamycin Kinase