The DOI matches its Crossref record; 1 of the 1 references it cites were found in a registry or an index.
Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells
Open accessAbstract
Gene transfer has potential as a once-only treatment that reduces viral load, preserves the immune system and avoids lifetime highly active antiretroviral therapy. This study, which is to our knowledge the first randomized, double-blind, placebo-controlled, phase 2 cell-delivered gene transfer clinical trial, was conducted in 74 HIV-1–infected adults who received a tat-vpr–specific anti-HIV ribozyme (OZ1) or placebo delivered in autologous CD34+ hematopoietic progenitor cells. There were no OZ1-related adverse events. There was no statistically significant difference in viral load between the OZ1 and placebo group at the primary end point (average at weeks 47 and 48), but time-weighted areas under the curve from weeks 40–48 and 40–100 were significantly lower in the OZ1 group. Throughout the 100 weeks, CD4+ lymphocyte counts were higher in the OZ1 group. This study indicates that cell-delivered gene transfer is safe and biologically active in individuals with HIV and can be developed as a conventional therapeutic product.
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Cites
High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR)
Schmidt, M., Schwarzwaelder, K., Bartholomae, C., Zaoui, K., Ball, C., Pilz, I., Braun, S., Glimm, H., & von Kalle, C. (2007) · Nature Methods · doi:10.1038/nmeth1103 · Cited by 328
Cited by
Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
Tebas, P., Stein, D., Tang, W. W., Frank, I., Wang, S. Q., Lee, G., Spratt, S. K., Surosky, R. T., Giedlin, M. A., Nichol, G., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1300662 · Cited by 1,357
Holt, N., Wang, J., Kim, K., Friedman, G., Wang, X., Taupin, V., Crooks, G. M., Kohn, D. B., Gregory, P. D., Holmes, M. C., et al. (2010) · Nature Biotechnology · doi:10.1038/nbt.1663 · Cited by 677
Mokany, E., Bone, S. M., Young, P. E., Doan, T. B., & Todd, A. V. (2010) · Journal of the American Chemical Society · doi:10.1021/ja9076777 · Cited by 275
Self-citationLi, L., Krymskaya, L., Wang, J., Henley, J., Rao, A., Cao, L.-F., Tran, C.-A., Torres-Coronado, M., Gardner, A., Gonzalez, N., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.65 · Cited by 181
Hofer, U., Henley, J. E., Exline, C. M., Mulhern, O., Lopez, E., & Cannon, P. M. (2013) · Journal of Infectious Diseases · doi:10.1093/infdis/jit382 · Cited by 43
Metadata
- Authors
- Mitsuyasu, R. T., Merigan, T. C., Carr, A., Zack, J. A., Winters, M. A., Workman, C., Bloch, M., Lalezari, J., Becker, S., Thornton, L., Akil, B., Khanlou, H., Finlayson, R., McFarlane, R., Smith, D. E., Garsia, R., Ma, D., Law, M., Murray, J. M., von Kalle, C., Ely, J. A., Patino, S. M., Knop, A. E., Wong, P., Todd, A. V., Haughton, M., Fuery, C., Macpherson, J. L., Symonds, G. P., Evans, L. A., Pond, S. M., & Cooper, D. A.
- Venue
- Nature Medicine
- Published
- 2009-02-15
- Volume
- 15
- Issue
- 3
- Pages
- 285-292
- ISSN
- 1078-8956 (ISSN-L)
- 1546-170X
- OpenAlex
- W2082371015
- Cited by
- 261
- References
- 1
- FWCI
- 15.15
- Type
- journal article
- Language
- en
- Open access
- green
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- The paper
- Mitsuyasu, R. T., Merigan, T. C., Carr, A., Zack, J. A., Winters, M. A., Workman, C., Bloch, M., Lalezari, J., Becker, S., Thornton, L., Akil, B., Khanlou, H., Finlayson, R., McFarlane, R., Smith, D. E., Garsia, R., Ma, D., Law, M., Murray, J. M., . . . Cooper, D. A. (2009). Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells. Nature Medicine. https://doi.org/10.1038/nm.1932
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
