Immune responses in liver-directed lentiviral gene therapy

Andrea Annoni, Kevin Goudy, Mahzad Akbarpour, Luigi Naldini, Maria Grazia Roncarolo

Research output: Contribution to journalArticle

Abstract

The use of lentiviral vectors (LV)s for in vivo gene therapy is an ideal platform for treating many types of disease. Since LVs can transduce a wide array of cells, support long-term gene expression, and be modified to enhance cell targeting, LVs are a powerful modality to deliver life-long therapeutic proteins. A major limitation facing the use of LVs for in vivo gene therapy is the induction of immune responses, which can reduce the transduction efficiency of LV, eliminate the transduced cells, and inhibit the effect of the therapeutic protein. LV strategies designed to restrict transgene expression to the liver to exploit its naturally tolerogenic properties have proven to significantly reduce the induction of pathogenic immune responses and increase therapeutic efficacy. In this review, we outline the immunological hurdles facing in vivo LV gene therapy and highlight the advantages and limitations of using liver-directed LV gene therapy.

Original languageEnglish
Pages (from-to)230-240
Number of pages11
JournalTranslational Research
Volume161
Issue number4
DOIs
Publication statusPublished - Apr 2013

ASJC Scopus subject areas

  • Medicine(all)
  • Biochemistry, medical
  • Public Health, Environmental and Occupational Health

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