Lapchak P A
Mov Disord. 1998;13 Suppl 1:49-54.
GDNF is a pleitropic neurotrophic factor which stimulates the dopaminergic phenotype in vitro and in vivo by way of activation of the GDNF/RET receptor complex. The pharmacologic profile of GDNF in two well-characterized animal models of Parkinson's disease suggests that the molecule may be useful in the treatment of neurodegenerative diseases involving dopaminergic dysfunction such as Parkinson's disease. This review summarizes the preclinical development path which was taken to develop GDNF as a novel therapeutic approach to treat Parkinson's disease based on GDNF's ability to regenerate dopamine neurons, including a description of the pharmacologic/biologic activities of GDNF. The overall aim will be to discuss these issues in the context of their potential therapeutic usefulness of GDNF to treat Parkinson's disease.
胶质细胞源性神经营养因子（GDNF）是一种多效性神经营养因子，它通过激活GDNF/RET受体复合物在体外和体内刺激多巴胺能表型。GDNF在两种特征明确的帕金森病动物模型中的药理学特性表明，该分子可能对治疗涉及多巴胺能功能障碍的神经退行性疾病（如帕金森病）有用。本综述总结了将GDNF开发为治疗帕金森病新疗法的临床前开发路径，该路径基于GDNF再生多巴胺神经元的能力，包括对GDNF药理/生物学活性的描述。总体目标是在GDNF治疗帕金森病的潜在治疗效用背景下讨论这些问题。
