Gene therapy is rapidly emerging as a powerful therapeutic strategy for a wide range of neurodegenerative disorders, including Alzheimer's disease (AD), Parkinson's disease (PD) and Huntington's disease (HD). Some early clinical trials have failed to achieve satisfactory therapeutic effects. Efforts to enhance effectiveness are now concentrating on three major fields: identification of new vectors, novel therapeutic targets, and reliable of delivery routes for transgenes. These approaches are being assessed closely in preclinical and clinical trials, which may ultimately provide powerful treatments for patients. Here, we discuss advances and challenges of gene therapy for neurodegenerative disorders, highlighting promising technologies, targets, and future prospects.

基因治疗正在迅速成为治疗多种神经退行性疾病的强大治疗策略，包括阿尔茨海默病 (AD)、帕金森病 (PD) 和亨廷顿病 (HD)。一些早期的临床试验未能取得满意的治疗效果。提高有效性的努力目前集中在三个主要领域：新载体的鉴定、新的治疗靶点以及可靠的转基因递送途径。这些方法正在临床前和临床试验中得到密切评估，最终可能为患者提供强大的治疗方法。在这里，我们讨论神经退行性疾病基因治疗的进展和挑战，重点介绍有前途的技术、目标和未来前景。