Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells.
Nat Med
; 15(3): 285-92, 2009 Mar.
Article
em En
| MEDLINE
| ID: mdl-19219022
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.
Texto completo:
1
Coleções:
01-internacional
Contexto em Saúde:
2_ODS3
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
/
Infecções por HIV
/
RNA Catalítico
/
HIV-1
/
Antígenos CD34
Tipo de estudo:
Clinical_trials
Limite:
Adult
/
Female
/
Humans
/
Male
Idioma:
En
Revista:
Nat Med
Ano de publicação:
2009
Tipo de documento:
Article