Gene therapy: a viable therapeutic strategy for Parkinson's disease?

被引:16
作者
Berry, Alexander L. [2 ]
Foltynie, Thomas [1 ]
机构
[1] UCL Inst Neurol, Sobell Dept Motor Neurosci, London WC1N 3BG, England
[2] UCL Inst Neurol, Dept Mol Neurosci, London WC1N 3BG, England
关键词
Parkinson's disease; Gene therapy; Basal ganglia; Non-motor; MIDBRAIN DOPAMINERGIC-NEURONS; LENTIVIRAL VECTOR DELIVERY; AMINO-ACID DECARBOXYLASE; RAT NIGROSTRIATAL SYSTEM; SLEEP BEHAVIOR DISORDER; GTP CYCLOHYDROLASE-I; NEUROTROPHIC FACTOR; NONMOTOR SYMPTOMS; OXIDATIVE STRESS; ALPHA-SYNUCLEIN;
D O I
10.1007/s00415-010-5796-9
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Gene therapy represents a potentially useful additional technique to ameliorate the motor symptoms of Parkinson's disease (PD), and the motor complications of its treatment. The neurodegenerative process itself, as well as the non-motor symptoms of PD, both remain less amenable to most of the current gene therapy approaches. This review presents an overview of the four gene therapies in phase I/II clinical trials, outlines some of the challenges they face, and proposes additional alternative strategies that might improve the clinical prospects of gene therapy for PD. In so doing, we hope to highlight the issue of the current absence of effective treatment for non-motor symptoms of PD and the potential of further candidate targets for gene therapy intervention that might improve upon this, for both specific individuals with genetic forms of PD as well as "sporadic" PD patients.
引用
收藏
页码:179 / 188
页数:10
相关论文
共 104 条
[1]   The epidemiology of dementia associated with Parkinson disease [J].
Aarsland, Dag ;
Kurz, Martin Wilhelm .
JOURNAL OF THE NEUROLOGICAL SCIENCES, 2010, 289 (1-2) :18-22
[2]   Neuropsychiatric Symptoms in Parkinson's Disease [J].
Aarsland, Dag ;
Marsh, Laura ;
Schrag, Anette .
MOVEMENT DISORDERS, 2009, 24 (15) :2175-2186
[3]   Memantine in patients with Parkinson's disease dementia or dementia with Lewy bodies: a double-blind, placebo-controlled, multicentre trial [J].
Aarsland, Dag ;
Ballard, Clive ;
Walker, Zuzana ;
Bostrom, Fredrik ;
Alves, Guido ;
Kossakowski, Katja ;
Leroi, Iracema ;
Pozo-Rodriguez, Francisco ;
Minthon, Lennart ;
Londos, Elisabet .
LANCET NEUROLOGY, 2009, 8 (07) :613-618
[4]  
Arnulf I, 2008, CURR OPIN NEUROL, V21, P472, DOI 10.1097/WCO.0b013e328305044d
[5]  
Azzouz M, 2002, J NEUROSCI, V22, P10302
[6]   Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC [J].
Bankiewicz, Krystof S. ;
Forsayeth, John ;
Eberling, Jamie L. ;
Sanchez-Pernaute, Rosario ;
Pivirotto, Philip ;
Bringas, John ;
Herscovitch, Peter ;
Carson, Richard E. ;
Eckelman, William ;
Reutter, Bryan ;
Cunningham, Janet .
MOLECULAR THERAPY, 2006, 14 (04) :564-570
[7]   Neuroprotective Effect of Long-term NDI1 Gene Expression in a Chronic Mouse Model of Parkinson Disorder [J].
Barber-Singh, Jennifer ;
Seo, Byoung Boo ;
Nakamaru-Ogiso, Eiko ;
Lau, Yuen-Sum ;
Matsuno-Yagi, Akemi ;
Yagi, Takao .
REJUVENATION RESEARCH, 2009, 12 (04) :259-267
[8]   Pathophysiology of levodopa-induced dyskinesia: Potential for new therapies [J].
Bezard, E ;
Brotchie, JM ;
Gross, CE .
NATURE REVIEWS NEUROSCIENCE, 2001, 2 (08) :577-588
[9]   Gene Therapy for Dopamine Replacement in Parkinson's Disease [J].
Bjorklund, Anders ;
Bjorklund, Tomas ;
Kirik, Deniz .
SCIENCE TRANSLATIONAL MEDICINE, 2009, 1 (02)
[10]   Scientific rationale for the development of gene therapy strategies for Parkinson's disease [J].
Bjorklund, Tomas ;
Kirik, Deniz .
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE, 2009, 1792 (07) :703-713