Regular ArticleIn VivoL-DOPA Production by Genetically Modified Primary Rat Fibroblast or 9L Gliosarcoma Cell Grafts via Coexpression of GTPcyclohydrolase I with Tyrosine Hydroxylase
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Cited by (63)
Design of a single AAV vector for coexpression of TH and GCH1 to establish continuous DOPA synthesis in a rat model of parkinson's disease
2012, Molecular TherapyCitation Excerpt :Together, these fractions constituted the whole forebrain and thus the total forebrain concentration could be calculated (Figure 5). As reported previously,13,14,15 the complete 6-OHDA lesion induced an almost 90% decrease in dorsal striatal BH4 levels (0.16 ± 0.02 pmol/mg compared to 1.4 ± 0.009 pmol/mg) (Figure 5a). The same pattern was observed in the ventral striatum and frontal cortex, although to a lesser extent.
Dopa-responsive dystonia
2011, Handbook of Clinical NeurologyCitation Excerpt :The loss of striatal TH protein with preservation in the SNc might be explained by BH4 controlling protein stability rather than expression (Furukawa et al., 1999). Leff et al. (1998) presented gene transfer data and suggested a role for stabilization of TH protein by co-expression of GCH-1 in vivo. Sumi-Ichinose et al. (2001) showed loss of TH protein but not of TH mRNA in the brains of BH4-deficient mice.
Gene therapy for dopamine replacement
2010, Progress in Brain ResearchCitation Excerpt :In two other studies, in vivo gene transfer, either recombinant adenoviral (Ad) or adeno-associated viral (AAV) vectors, were utilized to deliver the TH gene to the striatum. Corti and colleagues showed that animals that received Ad-TH vector displayed behavioral recovery linked to DOPA synthesis only after administration of significant amounts of BH4 peripherally (Corti et al., 1999), while Leff and collaborators used AAV vectors to co-express TH and GCH1 and achieved similar degree of recovery without administration of exogenous BH4 (Leff et al., 1998). The early enzyme replacement studies resulted in three major approaches for dopamine replacement in PD that are all based on gene therapy to render striatal neurons into dopamine- and DOPA-producing cells (Fig. 1).
Microdialysis in central nervous system disorders and their treatment
2008, Pharmacology Biochemistry and BehaviorControl of dopamine-secretion by Tet-Off system in an in vivo model of parkinsonian rat
2006, Brain ResearchCitation Excerpt :Serial measurement of DA in the CSF is very useful but at the same time we should be aware of the limitation of this estimation. Previous studies using either ex vivo approaches or viral vectors for local delivery of LD in the brain have demonstrated that the co-factor biopterin or the rate-limiting enzyme in the biopterin synthesis, GTP-cyclohydrolase-1, needs to be supplied for efficient LD production in addition to TH (Bencsics et al., 1996; Kirik et al., 2002; Leff et al., 1998). In our study, LD production of PC12 cells increased about 5-fold with human TH gene transfection.
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