OZ1

OZ1 is a gene therapy product developed as one of the first investigational gene therapies for HIV infection. It consists of an anti-HIV ribozyme delivered by an ex vivo transduction of autologous CD34+ hematopoietic progenitor cells with a retroviral vector encoding the ribozyme. The mechanism of action is to inhibit HIV replication in infected cells by cleaving HIV RNA, thus aiming to confer HIV resistance to the patient’s immune cells. It was primarily investigated for HIV/AIDS. OZ1 represented an early approach to RNA-based gene therapy targeting viral replication, but is not known to be currently in active clinical development or approved for use.

Other names

OZ1-containing CD34+ hematopoietic progenitor cellsOZ1-transduced CD34+ hematopoietic stem cellsOZ1-transduced hematopoietic progenitor cellsOZ-1-transduced hematopoietic progenitor cellsOZ 1-transduced hematopoietic progenitor cells