Gene therapy for Parkinson's disease: state-of-the-art treatments for neurodegenerative disease

被引:0
|
作者
Douglas, Michael R. [1 ,2 ]
机构
[1] Univ Birmingham, Sch Clin & Expt Med, Birmingham B15 2TT, W Midlands, England
[2] Dudley Grp Hosp NHS Fdn Trust, Dept Neurol, Russells Hall Hosp, Dudley DY1 2HQ, W Midlands, England
关键词
AAV-GAD; AAV-hAADC; AAV-NTN; adeno-associated virus; gene therapy; lentivirus; Parkinson's disease; CONVECTION-ENHANCED DELIVERY; AMINO-ACID DECARBOXYLASE; LENTIVIRAL VECTOR DELIVERY; MPTP-LESIONED PRIMATES; ALPHA-SYNUCLEIN; NEUROTROPHIC FACTOR; RAT MODEL; IN-VIVO; PHASE-I; ADENOASSOCIATED VIRUSES;
D O I
10.1586/ERN.13.58
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Pharmacological and surgical treatments offer symptomatic benefits to patients with Parkinson's disease; however, as the condition progresses, patients experience gradual worsening in symptom control, with the development of a range of disabling complications. In addition, none of the currently available therapies have convincingly shown disease-modifying effects - either in slowing or reversing the disease. These problems have led to extensive research into the possible use of gene therapy as a treatment for Parkinson's disease. Several treatments have reached human clinical trial stages, providing important information on the risks and benefits of this novel therapeutic approach, and the tantalizing promise of improved control of this currently incurable neurodegenerative disorder.
引用
收藏
页码:695 / 705
页数:11
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