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Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells
- Mitsuyasu, Ronald T;
- Merigan, Thomas C;
- Carr, Andrew;
- Zack, Jerome A;
- Winters, Mark A;
- Workman, Cassy;
- Bloch, Mark;
- Lalezari, Jacob;
- Becker, Stephen;
- Thornton, Lorna;
- Akil, Bisher;
- Khanlou, Homayoon;
- Finlayson, Robert;
- McFarlane, Robert;
- Smith, Don E;
- Garsia, Roger;
- Ma, David;
- Law, Matthew;
- Murray, John M;
- von Kalle, Christof;
- Ely, Julie A;
- Patino, Sharon M;
- Knop, Alison E;
- Wong, Philip;
- Todd, Alison V;
- Haughton, Margaret;
- Fuery, Caroline;
- Macpherson, Janet L;
- Symonds, Geoff P;
- Evans, Louise A;
- Pond, Susan M;
- Cooper, David A
Published Web Location
https://doi.org/10.1038/nm.1932Abstract
The first phase 2 gene therapy trial for HIV-1 has shown some promising signs. There's a long way to go before this would be a viable approach in people with HIV—this trial did not show a statistically significant difference in viral load at the primary end point–but other analyses did reveal that the gene therapy seemed to have a modest, but statistically significant, effect at reducing viral load in the treated subjects versus the placebo arm. The study also provides some clues about what to improve in the future.
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