hMSC-GDNF

hMSC-GDNF refers to **human mesenchymal stem cells (hMSCs) engineered to express and/or secrete glial cell line-derived neurotrophic factor (GDNF)**. This therapeutic product combines cell therapy (using hMSCs, typically derived from adipose tissue or bone marrow) with gene therapy or protein loading to deliver GDNF either via transgenic expression (e.g., lentiviral vector, Tet-On regulatable system) or by loading cells with GDNF protein prior to transplantation. The principal mechanism is neuroprotection and neurorestoration—GDNF promotes survival, maintenance, and differentiation of dopaminergic neurons and other neural cell types. The delivery vehicle (hMSC) itself may also provide trophic support, enhance cell survival, reduce local inflammation, and assist tissue regeneration. This strategy is under investigation primarily for neurodegenerative diseases, especially Parkinson’s disease and ALS, where direct delivery of GDNF is believed to be beneficial but has shown technical limits and mixed results in direct protein administration. The system can be regulated for GDNF expression (e.g., Tet-On) to avoid the adverse effects of continuous overexpression. Studies have demonstrated induction of neuroprotective effects in in vitro and animal models, including protection of dopaminergic neurons and improved motor function in rodent models of Parkinson’s disease[1][2][4].

Other names

human mesenchymal stem cells expressing glial cell line-derived neurotrophic factor