granulocyte-macrophage colony-stimulating factor mRNA

Granulocyte-macrophage colony-stimulating factor mRNA is an **mRNA-based drug** encoding the protein granulocyte-macrophage colony-stimulating factor (GM-CSF, also known as CSF2). Unlike recombinant GM-CSF protein drugs, this modality uses mRNA delivered by lipid nanoparticle (LNP) formulation to induce endogenous expression of GM-CSF protein following cellular uptake. The GM-CSF protein is a hematopoietic cytokine that promotes the differentiation and proliferation of granulocytes, monocytes, macrophages, and dendritic cells, and acts as an immune modulator in both innate and adaptive immunity. The mRNA platform aims to improve pharmacokinetic profiles such as half-life and reduce the need for repeated protein dosing. Preclinical studies have demonstrated that GM-CSF mRNA therapy, particularly via LNP, has neuroprotective and anti-inflammatory effects, including expansion of regulatory T cells (Tregs) and suppression of pro-inflammatory responses in neurodegenerative disease models like Parkinson’s disease.

Other names

granulocyte-macrophage colony-stimulating factor mRNAGM-CSF mRNACsf2 mRNACsf-2 mRNACsf 2 mRNA