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shRNA-modified CD34+ cells
- Development stage
- Phase 1
- Lead developer
- Boston Children's Hospital
- Modality
- Cell Therapies, Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
shRNA-modified CD34+ cells refers to an autologous cell therapy and gene therapy product consisting of hematopoietic stem cells (HSCs) genetically engineered to resist HIV-1 infection. In the specific program led by City of Hope and Benitec Biopharma (NCT01734850), CD34+ cells are transduced with a lentiviral vector (rHIV7-shI-TAR-CCR5RZ) that expresses three distinct RNA-based inhibitors: a short hairpin RNA (shRNA) targeting the HIV-1 tat and rev genes, a TAR decoy to sequester the viral Tat protein, and a ribozyme targeting the CCR5 coreceptor. Once infused back into the patient, these modified cells are intended to engraft in the bone marrow and give rise to a protected population of T cells and macrophages that are resistant to viral entry and replication, potentially providing a long-term functional cure for HIV/AIDS.
- Other names
- Multiplexed shRNA-modified CD34+ cellsLentiviral-transduced CD34+ cells
02
Targets
BCL11A (B-cell CLL/lymphoma 11A)
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