2008
DOI: 10.1016/j.nurt.2008.01.005
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Advances in Gene Therapy for Movement Disorders

Abstract: Summary:After nearly 20 years of preclinical experimentation with various gene delivery approaches in animal models of Parkinson's disease (PD), clinical trials are finally underway. The risk/benefit ratio for these procedures is now generally considered acceptable under approved protocols. The current vehicle for gene delivery to the human brain is recombinant adeno-associated viral vector, which is nonpathogenic and non-self-amplifying. Candidate genes tested in PD patients encode 1) glutamic acid decarboxyl… Show more

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Cited by 24 publications
(10 citation statements)
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“…Till now, gene therapy of PD using therapeutic genes encoding glial cell line-derived neurotrophic factor (GDNF) family has been extensively studied in animals, even advanced to clinical testing [14,15]. GDNF is generally believed to possess most potent trophic effects on dopaminergic neurons [16].…”
Section: Introductionmentioning
confidence: 99%
“…Till now, gene therapy of PD using therapeutic genes encoding glial cell line-derived neurotrophic factor (GDNF) family has been extensively studied in animals, even advanced to clinical testing [14,15]. GDNF is generally believed to possess most potent trophic effects on dopaminergic neurons [16].…”
Section: Introductionmentioning
confidence: 99%
“…Therefore, gene therapy of PD using therapeutic genes encoding GDNF family has been extensively studied in animals, even advanced to clinical testing (Hong et al, 2008;Mochizuki et al, 2008). Zhang and Pardridge (2009) reported that rats with experimental PD are treated with intravenous GDNF plasmid DNA (designated pTHproGDNF) and nonviral gene therapy using Trojan horse liposomes targeted with a MAb to the rat TfR.…”
Section: Gene Therapymentioning
confidence: 99%
“…(106, 108–113) One approach focuses on increasing dopamine production via direct delivery of genes involved in neurotransmitter synthesis while a second method is designed to change the neuronal phenotype bypassing the need for dopamine, both approaches should the ameliorate symptoms associated with PD. These therapies are also intended to delay development of end-stage disease, an important accomplishment in a progressive age-related disorder such as PD.…”
Section: Parkinson’s Disease Gene Therapy Clinical Trialsmentioning
confidence: 99%