HIV sequence variation associated with env antisense adoptive T-cell therapy in the hNSG mouse model

Mol Ther. 2010 Apr;18(4):803-11. doi: 10.1038/mt.2009.316. Epub 2010 Jan 26.

Abstract

The first use of lentiviral vectors in humans involved transduction of mature T-cells with an human immunodeficiency virus (HIV)-derived env antisense (envAS) vector to protect cells from HIV infection. In that study, only a minority of the patient T-cell population could be gene-modified, raising the question of whether the altered cells could affect replicating HIV populations. We investigated this using humanized NOD/SCID IL-2Rgamma(null) (hNSG) mice reconstituted with approximately 4-11% envAS-modified human T-cells. Mice were challenged with HIV-1(NL4-3), which has an env perfectly complementary to envAS, or with HIV-1(BaL), which has a divergent env. No differences were seen in viral titer between mice that received envAS-modified cells and control mice that did not. Using 454/Roche pyrosequencing, we analyzed the mutational spectrum in HIV populations in serum-from 33 mice we recovered 84,074 total reads comprising 31,290 unique sequence variants. We found enrichment of A-to-G transitions and deletions in envAS-treated mice, paralleling a previous tissue culture study where most target cells contained envAS, even though minority of cells were envAS-modified here. Unexpectedly, this enrichment was only detected after the challenge with HIV-1(BaL), where the viral genome would form an imperfect duplex with envAS, and not HIV-1(NL4-3), where a perfectly matched duplex would form.

Publication types

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

MeSH terms

  • Animals
  • Base Sequence
  • Genetic Variation
  • HIV Infections / therapy*
  • HIV Infections / virology
  • HIV-1 / genetics*
  • Humans
  • Immunotherapy, Adoptive*
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • RNA, Antisense / genetics*
  • RNA, Viral / genetics
  • T-Lymphocytes / immunology
  • T-Lymphocytes / transplantation*
  • Transduction, Genetic
  • Virus Replication / genetics
  • env Gene Products, Human Immunodeficiency Virus / genetics*

Substances

  • RNA, Antisense
  • RNA, Viral
  • env Gene Products, Human Immunodeficiency Virus