anti-HIV-1 tat ribozyme

anti-HIV-1 tat ribozyme is an *ex vivo* gene therapy product developed by Ribozyme Pharmaceuticals (later Sirna Therapeutics) for the treatment of HIV infection and HIV-related non-Hodgkin's lymphoma. The therapy utilizes a retroviral vector (typically LNL6) to deliver a hammerhead ribozyme transgene specifically designed to target and catalytically cleave the HIV-1 *tat* mRNA. By destroying the *tat* transcript, the therapy prevents the synthesis of the Tat protein, a critical transactivator required for viral gene expression and replication. The construct also includes a neomycin resistance gene (*neo*) as a selectable marker. In clinical trials, autologous CD34+ hematopoietic stem cells were transduced with the vector and re-infused into patients, with the goal of creating a reservoir of immune cells resistant to HIV-1 infection.

Other names

L-TR + Tat-neo-Ribozyomeanti-HIV-1 tat hammerhead ribozymeanti-HIV1 tat hammerhead ribozymeanti-HIV 1 tat hammerhead ribozymeribozyme-transduced autologous stem cell transplantation