Lentiviral Ciliary Neurotrophic Factor (LV-CNTF)

Lentiviral Ciliary Neurotrophic Factor (LV-CNTF) is a gene therapy approach that utilizes a lentiviral vector to deliver the gene encoding Ciliary Neurotrophic Factor (CNTF). CNTF is a neurotrophic cytokine that plays a crucial role in promoting the survival, differentiation, and function of neurons, particularly photoreceptors and retinal ganglion cells. Upon successful gene transfer and expression, the secreted CNTF protein binds to its specific receptor complex, which consists of Ciliary Neurotrophic Factor receptor alpha (CNTFRα), Leukemia inhibitory factor receptor beta (LIFRβ), and Glycoprotein 130 (gp130). This binding event activates intracellular signaling pathways, primarily the Janus kinase/signal transducer and activator of transcription (JAK/STAT) pathway, specifically STAT3, and the MEK-ERK pathway, leading to neuroprotective effects. LV-CNTF has been investigated in preclinical studies for its potential to treat various retinal degenerative diseases by preserving retinal cells.

Other names

CNTF gene therapy (lentiviral vector)