granulocyte colony-stimulating factor gene therapy

CHS Pharma is developing a gene therapy candidate that delivers the gene for granulocyte colony-stimulating factor (G-CSF) for the treatment of ischemic stroke and Alzheimer's disease. G-CSF is a glycoprotein growth factor that traditionally stimulates the bone marrow to produce granulocytes and stem cells. In the central nervous system, G-CSF acts as a neurotrophic factor, promoting neurogenesis, increasing neuroplasticity, and counteracting apoptosis. This gene therapy approach is designed to provide sustained endogenous expression of G-CSF, overcoming the limitations of the protein's short 4-hour plasma half-life. The therapy targets the G-CSF receptor (G-CSFR), which is expressed on both hematopoietic precursor cells and neurons. It is currently in the preclinical stage of development.

Other names

G-CSF gene therapyGCSF gene therapygranulocyte colony-stimulating factor gene therapy