Gene Therapy of Parkinson Disease Model Rat by Direct Injection of Plasmid DNA–Lipofectin Complex

Lipofectin-mediated gene transfer was used to introduce plasmid harboring the tyrosine hydroxylase (TH) gene into the striatum of rats with lesions of the nigrostriatal pathway. The rotational asymmetry of Parkinson disease model rat was reduced quickly and significantly, suggesting that plasmid–DNA-transfected brain cells can generate l-dopa locally in the striatum in quantities sufficient to compensate partially for the loss of intrinsic striatal dopaminergic input. Immunohistochemical staining and reverse transcription polymerase chain reaction (RT-PCR) also confirm that striatal cells can express exogenous TH gene. Such in vivo plasmid DNA transfer strategy may be useful in other neurologic disease therapy, especially acute brain insults. Parkinson disease (PD) is an useful model for the study of neurologic gene therapy, and some ex vivo strategies have been evaluated for PD treatment Cao et al. examine the in vivo lipofectin-mediated gene therapy in 6-hydroxy-dopamine-lesioned rats.