lentiviral tyrosine hydroxylase-transduced bone marrow-derived mesenchymal stem cells

Lentiviral tyrosine hydroxylase-transduced bone marrow-derived mesenchymal stem cells (LV-TH-BMSCs) are a genetically engineered cell therapy developed for the treatment of Parkinson's disease. This therapy involves isolating mesenchymal stem cells from bone marrow and using a lentiviral vector to introduce the human tyrosine hydroxylase (TH) gene, which encodes the rate-limiting enzyme for dopamine synthesis. Once transplanted into the brain—specifically the striatum or medial forebrain bundle—these cells act as a biological pump to continuously produce L-DOPA, which is then converted into dopamine by endogenous or co-transplanted enzymes. Beyond neurotransmitter replacement, the BMSCs provide a neuroprotective microenvironment by secreting anti-inflammatory cytokines and neurotrophic factors, thereby reducing neuroinflammation and promoting the survival of host dopaminergic neurons. Preclinical studies in rat models have demonstrated that these cells can survive long-term, differentiate into functional neurons, and significantly improve motor deficits.

Other names

TH-transduced BMSCsLV-TH BMSCstyrosine hydroxylase-engineered bone marrow mesenchymal stem cells