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gene therapy for Parkinson's disease
Procedure: (human)
- adenovirus vector containing glutamic acid decarboxylase gene (GAD) injected into the subthalamic nucleus
Clinical significance:
- improvement in motor function
Adverse effects:
- no adverse effects
Related
Parkinson's disease (paralysis agitans, PD)
General
gene therapy
References
- Kaplitt MG, Feigin A, Tang C, Fitzsimons HL, Mattis P,
Lawlor PA, Bland RJ, Young D, Strybing K, Eidelberg D, During MJ.
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.
Lancet. 2007 Jun 23;369(9579):2097-105.
PMID: 17586305
- Feigin A, Kaplitt MG, Tang C, Lin T, Mattis P, Dhawan V,
During MJ, Eidelberg D.
Modulation of metabolic brain networks after subthalamic
gene therapy for Parkinson's disease.
Proc Natl Acad Sci U S A. 2007 Dec 4;104(49):19559-64.
Epub 2007 Nov 27.
PMID: 18042721