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. and Cooper, David A. (2009) Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34 + cells. Nature Medicine, 15 3: 285-292. doi:10.1038/nm.1932

Author 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.
Title Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34 + cells
Formatted title
Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34 + cells
Journal name Nature Medicine   Check publisher's open access policy
ISSN 1078-8956
Publication date 2009-03-01
Sub-type Article (original research)
DOI 10.1038/nm.1932
Open Access Status Not yet assessed
Volume 15
Issue 3
Start page 285
End page 292
Total pages 8
Place of publication New York, United States
Publisher Nature Publishing Group
Language eng
Formatted abstract
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.
Q-Index Code C1
Q-Index Status Provisional Code
Institutional Status Non-UQ

Document type: Journal Article
Sub-type: Article (original research)
Collection: School of Public Health Publications
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Citation counts: TR Web of Science Citation Count  Cited 158 times in Thomson Reuters Web of Science Article | Citations
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Created: Mon, 28 Mar 2011, 21:40:46 EST by Ms Margaret Haughton on behalf of School of Public Health