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7shRNA-modified autologous hematopoietic stem cells
- Development stage
- Unknown
- Lead developer
- Hangzhou First People's Hospital
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Stem Cell Therapies → Cell Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
7shRNA-modified autologous hematopoietic stem cells is an experimental cell and gene therapy being developed for the treatment of HIV infection complicated by lymphoma. The therapy involves the extraction of a patient's own hematopoietic stem cells, which are then genetically modified using a lentiviral vector to stably express seven distinct short hairpin RNAs (7shRNA). These shRNAs are designed to target multiple conserved regions of the HIV-1 genome to inhibit viral replication and prevent the infection of new cells. The modified cells are then transplanted back into the patient following chemotherapy. This approach aims to provide a functional cure for HIV by creating a reservoir of HIV-resistant immune cells while simultaneously treating the underlying lymphoma through autologous stem cell transplantation.
- Other names
- 7shRNA-Modified Autologous Stem Cell Transplantation7shRNA-modified autologous stem cells
02
Targets
HIV-1 RNA (HIV-1 RNA genome)
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