ArticleProceedings of the National Academy of Sciences of the United States of America2007
Stable reduction of CCR5 by RNAi through hematopoietic stem cell transplant in non-human primates.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 90 papers.
What it found
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
90 citing papers in PubMed, 149 citations in OpenAlex.
- Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells.Nature medicine · 2009Trial
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- Optimal delivery of RNA interference by viral vectors for cancer therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Review
- Potent dual block to HIV-1 infection using lentiviral vectors expressing fusion inhibitor peptide mC46- and Vif-resistant APOBEC3G.Molecular therapy. Nucleic acids · 2023Article
- Efficient derivation of chimeric-antigen receptor-modified TFrontiers in immunology · 2022Article
- Preclinical Development and Clinical-Scale Manufacturing of HIV Gag-Specific, LentivirusModified CD4 T Cells for HIV Functional Cure.Molecular therapy. Methods & clinical development · 2020Article
- CRISPR therapy towards an HIV cure.Briefings in functional genomics · 2020Article
- Gene Therapy Approaches to Functional Cure and Protection of Hematopoietic Potential in HIV Infection.Pharmaceutics · 2019Review
- Modeling Anti-HIV-1 HSPC-Based Gene Therapy in Humanized Mice Previously Infected with HIV-1.Molecular therapy. Methods & clinical development · 2018Article
- Modern biotechnology-based therapeutic approaches against HIV infection.Biomedical reports · 2017Article
- Purging Exhausted Virus-Specific CD8 T Cell Phenotypes by Somatic Cell Reprogramming.AIDS research and human retroviruses · 2017Article
- Article
- Recent advances in RNAi-based strategies for therapy and prevention of HIV-1/AIDS.Advanced drug delivery reviews · 2016Review
- Review
- Stem cell-based therapies for HIV/AIDS.Advanced drug delivery reviews · 2016Review
- Development of Lentiviral Vectors Simultaneously Expressing Multiple siRNAs Against CCR5, vif and tat/rev Genes for an HIV-1 Gene Therapy Approach.Molecular therapy. Nucleic acids · 2016Article
- Mechanistic Models Predict Efficacy of CCR5-Deficient Stem Cell Transplants in HIV Patient Populations.CPT: pharmacometrics & systems pharmacology · 2016Article
- The feasibility of incorporating Vpx into lentiviral gene therapy vectors.Molecular therapy. Methods & clinical development · 2016Article
- Multilineage polyclonal engraftment of Cal-1 gene-modified cells and in vivo selection after SHIV infection in a nonhuman primate model of AIDS.Molecular therapy. Methods & clinical development · 2016Article
30 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 5 institutions in 2 countries.
Funding
Abstract
RNAi is a powerful method for suppressing gene expression that has tremendous potential for therapeutic applications. However, because endogenous RNAi plays a role in normal cellular functions, delivery and expression of siRNAs must be balanced with safety. Here we report successful stable expression in primates of siRNAs directed to chemokine (c-c motif) receptor 5 (CCR5) introduced through CD34+ hematopoietic stem/progenitor cell transplant. After hematopoietic reconstitution, to date 14 months after transplant, we observe stably marked lymphocytes expressing siRNAs and consistent down-regulation of chemokine (c-c motif) receptor 5 expression. The marked cells are less susceptible to simian immunodeficiency virus infection ex vivo. These studies provide a successful demonstration that siRNAs can be used together with hematopoietic stem cell transplant to stably modulate gene expression in primates and potentially treat blood diseases such as HIV-1.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.