TYF-CGD-modified autologous hematopoietic stem cells

TYF-CGD-modified autologous hematopoietic stem cells is an ex vivo gene therapy candidate developed by the Shenzhen Geno-Immune Medical Institute for the treatment of Chronic Granulomatous Disease (CGD). The therapy involves the harvest of a patient's own CD34+ hematopoietic stem cells (HSCs), which are then genetically modified ex vivo using the TYF self-inactivating lentiviral vector. This vector is designed to deliver a functional copy of either the CYBB gene (encoding the gp91phox subunit) for X-linked CGD or the NCF1 gene (encoding the p47phox subunit) for autosomal recessive CGD. Chronic Granulomatous Disease is characterized by mutations in the NADPH oxidase complex, preventing phagocytes from producing the reactive oxygen species (ROS) necessary for killing pathogens. Once the modified cells are re-infused into the patient following conditioning, they engraft in the bone marrow and differentiate into functional myeloid cells, thereby restoring the phagocytic respiratory burst and protecting the patient from recurrent life-threatening infections.

Other names

Lentiviral TYF-CGD-modified autologous stem cellsTYF-CGD gene therapyTYF-CGD lentiviral gene therapy