Table 1 : Genetic therapies against HIV : Nature Biotechnology

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From the following article

Genetic therapies against HIV

John J Rossi, Carl H June & Donald B Kohn

Nature Biotechnology 25, 1444 - 1454 (2007) Published online: 7 December 2007

doi:10.1038/nbt1367

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Table 1. Completed and ongoing gene therapy trials for HIV

Figures and tables index

Protocol description	Phase	Status	Payload	Cellular vehicle	Transfer vector	References
T cells
A randomized study of HIV-specific T-cell gene therapy in subjects with undetectable plasma viremia on combination antiretroviral therapy	2	Completed	CD4 receptor coupled with the CD3 signaling chain	Autologous CD4⁺ and CD8⁺ T cells; (3) repeat doses	Murine retrovirus	95
Evaluation of safety, tolerability and persistence of escalating and repeat doses of genetically modified syngeneic CD8⁺ or CD4⁺/CD8⁺ cells	1–2	Completed	CD4 receptor coupled with the CD3 signaling chain	Syngeneic CD8⁺ or CD4⁺/CD8⁺ cells; single or multiple doses	Murine retrovirus	93
Evaluation of safety, tolerability and tissue trafficking of a single dose of genetically modified autologous CD4⁺ and CD8⁺ cells.	1–2	Completed	CD4 receptor coupled with the CD3 signaling chain	Autologous CD4⁺ and CD8⁺ cells; single dose	Murine retrovirus	94
Evaluation of safety and tolerability of a single infusion of autologous CD4⁺ T cells modified with a dominant-negative anti-HIV gene	1–2	Completed	Rev M10	Autologous CD4⁺ cells; single dose	Gold particles / murine retrovirus	41,74
A marker study of therapeutically transduced CD4⁺ peripheral blood lymphocytes in HIV-discordant identical twins	1	Completed	Anti–HIV-1 tat ribozyme (Rz2)	Syngeneic CD4⁺ cells (twin study); single dose	Murine retrovirus	76
Evaluation of safety and tolerability of multiple infusions of syngeneic CD4⁺ lymphocytes modified with anti-HIV genes	1	Completed	Trans-dominant rev and/or trans-dominant rev with TAR antisense	Syngeneic CD4⁺ cells (twin study); two doses	Murine retrovirus	75
Evaluation of safety and tolerability of ribozyme gene therapy of HIV-1 infection	1	Completed	Anti–HIV-1 Rz to the U5 leader sequence	Autologous CD4⁺ cells; single dose	Murine retrovirus	128
Evaluation of safety and tolerability of a single dose of autologous T cells transduced with VRX496 in HIV-positive patient subjects	1–2	Completed	Anti–HIV-1 antisense against the envelope gene	Autologous CD4⁺ T cells; single dose	HIV-derived lentivirus, conditionally replicating	9
Evaluation of safety, tolerability and antiviral effects of autologous CD4⁺ T cells expressing the HIV fusion inhibitor M87	1	Completed	gp41 fusion peptide inhibitor	Autologous CD4⁺ T cells	Murine retrovirus	62
An open-label, multicenter study to evaluate the safety, tolerability and biological activity of repeated doses of autologous T cells transduced with VRX496 in HIV-positive subjects	2	Ongoing	Anti HIV-1 antisense against the envelope gene	Autologous CD4⁺T cells; (4 or 8) repeat doses	HIV-derived lentivirus, conditionally replicating
An open-label, single-center study to evaluate the tolerability, trafficking and therapeutic effects of repeated doses of autologous T cells transduced with VRX496 in HIV-infected subjects	1–2	Ongoing	Anti HIV-1 antisense against the envelope gene	Autologous CD4⁺ T cells; (6) repeat doses	HIV-derived lentivirus, conditionally replicating
HS cells
Nonmyeloablative conditioning followed by transplantation of genetically modified HLA-matched peripheral blood progenitor cells for hematological malignancies in persons with acquired immunodeficiency syndrome	1	Completed	Trans-dominant Rev	Autologous CD34⁺ cells isolated from mobilized peripheral blood	Murine retrovirus	129
Evaluation of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34⁺ cells from the bone marrow of HIV-1–infected children	1	Completed	RRE decoy	Autologous CD34⁺ bone marrow cells	Murine retrovirus	14
Evaluation of safety, tolerability and persistence of transplantation with autologous bone marrow transduced with a retroviral vector expressing dominant-negative Rev or a control gene	1	Completed	Trans-dominant Rev	Autologous CD34⁺ bone marrow cells	Murine retrovirus	120
Evaluation of safety and tolerability of autologous CD34⁺ hematopoietic progenitor cells transduced with an anti-HIV ribozyme	1	Completed	Anti–HIV-1 tat ribozyme (Rz2)	Autologous CD34⁺ cells isolated from mobilized peripheral blood	Murine retrovirus	77,119
A randomized, double-blind, controlled trial to evaluate the safety and efficacy of autologous CD34⁺ hematopoietic progenitor cells transduced with placebo or an anti–HIV-1 ribozyme (OZ1) in patients with HIV-1 infection	2	Ongoing	Anti-HIV ribozyme OZ1	Autologous CD34⁺ cells isolated from mobilized peripheral blood	Murine retrovirus	http://clinicaltrials.gov/show/NCT00074997
A pilot study of safety and feasibility of stem-cell therapy for AIDS lymphoma using stem cells treated with a lentivirus vector encoding multiple anti-HIV RNAs	1	Ongoing	Triple combination vector co-expressing an anti tat/rev shRNA, a nucleolar localizing TAR decoy and an anti-CCR5 ribozyme in a single vector backbone	Autologous CD34⁺ HS cells	HIV-derived lentivirus	http://clinicaltrials.coh.org/specific_result.aspx? dise=0&category=1023&age=&protgroup= &phase=&gender=&keyword1=&frmexact

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