Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial
Tài liệu tham khảo
Somia, 2000, Gene therapy: trials and tribulations, Nat Rev Genet, 1, 91, 10.1038/35038533
Manno, 2006, Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response, Nat Med, 12, 342, 10.1038/nm1358
Pulkkanen, 2005, Gene therapy for malignant glioma: current clinical status, Mol Ther, 12, 585, 10.1016/j.ymthe.2005.07.357
McPhee, 2006, Immune responses to AAV in a phase I study for Canavan disease, J Gene Med, 8, 577, 10.1002/jgm.885
Tuszynski, 2005, A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease, Nat Med, 11, 551, 10.1038/nm1239
Obeso, 2000, Pathophysiologic basis of surgery for Parkinson's disease, Neurology, 55, S7
Wichmann, 2003, Pathophysiology of Parkinson's disease: the MPTP primate model of the human disorder, Ann N Y Acad Sci, 991, 199, 10.1111/j.1749-6632.2003.tb07477.x
Lang, 1998, Parkinson's disease. First of two parts, N Engl J Med, 339, 1044, 10.1056/NEJM199810083391506
Nutt, 2005, Clinical practice. Diagnosis and initial management of Parkinson's disease, N Engl J Med, 353, 1021, 10.1056/NEJMcp043908
Erlander, 1991, Two genes encode distinct glutamate decarboxylases, Neuron, 7, 91, 10.1016/0896-6273(91)90077-D
Hamani, 2004, The subthalamic nucleus in the context of movement disorders, Brain, 127, 4, 10.1093/brain/awh029
Emborg, 2007, Subthalamic glutamic acid decarboxylase gene therapy: changes in motor function and cortical metabolism, J Cereb Blood Flow Metab, 27, 501, 10.1038/sj.jcbfm.9600364
Kaplitt, 1994, Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain, Nat Genet, 8, 148, 10.1038/ng1094-148
Lee, 2005, Enhanced expression of glutamate decarboxylase 65 improves symptoms of rat parkinsonian models, Gene Ther, 12, 1215, 10.1038/sj.gt.3302520
Luo, 2002, Subthalamic GAD gene therapy in a Parkinson's disease rat model, Science, 298, 425, 10.1126/science.1074549
Hoehn, 1992, The natural history of Parkinson's disease in the pre-levodopa and post-levodopa eras, Neurol Clin, 10, 331, 10.1016/S0733-8619(18)30213-5
Metman, 2004, Test-retest reliability of UPDRS-III, dyskinesia scales, and timed motor tests in patients with advanced Parkinson's disease: an argument against multiple baseline assessments, Mov Disord, 19, 1079, 10.1002/mds.20101
Schwab R, England A. Projection technique for evaluating surgery in Parkinson's disease. Third symposium on Parkinson's disease 1969, Edinburgh: 152–157.
Asanuma, 2006, Network modulation in the treatment of Parkinson's disease, Brain, 129, 2667, 10.1093/brain/awl162
Hobson, 2002, Excessive daytime sleepiness and sudden-onset sleep in Parkinson disease: a survey by the Canadian Movement Disorders Group, JAMA, 287, 455, 10.1001/jama.287.4.455
Chirmule, 1999, Immune responses to adenovirus and adeno-associated virus in humans, Gene Ther, 6, 1574, 10.1038/sj.gt.3300994
Palombo, 1990, Local cerebral glucose utilization in monkeys with hemiparkinsonism induced by intracarotid infusion of the neurotoxin MPTP, J Neurosci, 10, 860, 10.1523/JNEUROSCI.10-03-00860.1990
Herzog, 1997, Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus, Proc Natl Acad Sci USA, 94, 5804, 10.1073/pnas.94.11.5804
Eidelberg, 1997, Metabolic correlates of pallidal neuronal activity in Parkinson's disease, Brain, 120, 1315, 10.1093/brain/120.8.1315
Eidelberg, 1996, Regional metabolic correlates of surgical outcome following unilateral pallidotomy for Parkinson's disease, Ann Neurol, 39, 450, 10.1002/ana.410390407
Su, 2001, Metabolic changes following subthalamotomy for advanced Parkinson's disease, Ann Neurol, 50, 514, 10.1002/ana.1232
Fukuda, 2001, Networks mediating the clinical effects of pallidal brain stimulation for Parkinson's disease: a PET study of resting-state glucose metabolism, Brain, 124, 1601, 10.1093/brain/124.8.1601
Oh, 2002, Long-term hardware-related complications of deep brain stimulation, Neurosurgery, 50, 1268
Kleiner-Fisman, 2006, Subthalamic nucleus deep brain stimulation: summary and meta-analysis of outcomes, Mov Disord, 21, S290, 10.1002/mds.20962
Deuschl, 2006, A randomized trial of deep-brain stimulation for Parkinson's disease, N Engl J Med, 355, 896, 10.1056/NEJMoa060281
Simuni, 2002, Bilateral stimulation of the subthalamic nucleus in patients with Parkinson disease: a study of efficacy and safety, J Neurosurg, 96, 666, 10.3171/jns.2002.96.4.0666