Gene Therapy for AIDS Using Retroviral Mediated Gene Transfer to Deliver HIV-1 Anti-Sense TAR and Transdominant Rev Protein Genes to Syngeneic Lymphocytes in HIV-1 Infected Identical Twins
This phase I/II pilot study will evaluate the safety, relative survival, and potential
efficacy of infusions of activated, genetically engineered, syngeneic CD4+ T lymphocytes
obtained from HIV-1 seronegative identical twins. T cells from each seronegative twin will
be obtained by apheresis, enriched for CD4+ cells, induced to polyclonal proliferation with
anti-CD3 and rIL-2 stimulation, divided into aliquots which will then be individually
transduced with a control retroviral vector and up to two additional retroviral vectors
containing potentially therapeutic genes (antisense TAR and/or transdominant Rev). These
engineered T cell populations will be expanded 10-1,000 fold in numbers during 1-2 weeks of
culture, and then will be infused into the seropositive twins. The relative survival of the
uniquely engineered T cell populations will be monitored by vector-specific PCR, while the
recipients' functional immune status is monitored by standard in vitro and in vivo testing
protocols. A total of up to 4 cycles of treatment may be given using identical or different
combinations of control and anti-HIV-1 retroviral vectors.
Interventional
Endpoint Classification: Safety Study, Primary Purpose: Treatment
United States: Federal Government
960051
NCT00001535
March 1996
March 2002
Name | Location |
---|---|
National Human Genome Research Institute (NHGRI) | Bethesda, Maryland 20892 |