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- Title
Secreted antiviral entry inhibitory (SAVE) peptides for gene therapy of HIV infection.
- Authors
Egerer, Lisa; Volk, Andreas; Kahle, Joerg; Kimpel, Janine; Brauer, Frances; Hermann, Felix G; von Laer, Dorothee
- Abstract
Gene therapeutic strategies for human immunodeficiency virus type 1 (HIV-1) infection could potentially overcome the limitations of standard antiretroviral drug therapy (ART). However, in none of the clinical gene therapy trials published to date, therapeutic levels of genetic protection have been achieved in the target cell population for HIV-1. To improve systemic antiviral efficacy, C peptides, which are efficient inhibitors of HIV-1 entry, were engineered for high-level secretion by genetically modified cells. The size restrictions for efficient peptide export through the secretory pathway were overcome by expressing the C peptides as concatemers, which were processed into monomers by furin protease cleavage. These secreted antiviral entry inhibitory (SAVE) peptides mediated a substantial protective bystander effect on neighboring nonmodified cells, thus suppressing virus replication even if only a small fraction of cells was genetically modified. Accordingly, these SAVE peptides may provide a strong benefit to AIDS patients in future, and, if applied by direct in vivo gene delivery, could present an effective alternative to antiretroviral drug regimen.
- Publication
Molecular therapy : the journal of the American Society of Gene Therapy, 2011, Vol 19, Issue 7, p1236
- ISSN
1525-0024
- Publication type
Journal Article
- DOI
10.1038/mt.2011.30