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R#23 (GPP): The diagnostic L-Dopa trial should be limited to children with features suggestive of dopa-responsive dystonia such as lower limb dystonia with diurnal variation and absent HPA

HEK293T cells were transfected with 2 g pX330-SHARP 3gRNA and 4 g plasmid repair template comprising EGFP-P2A-puromycin and flanking 1 kb homology arms for SHARP-GFP or 2 g pX330-SHARP 3gRNA + 2 g pX330-SHARP SPOC gRNA to excise the region encoding SPOC and 4 g plasmid repair template for SHARP SPOC-GFP

Remember that clinical trials failed to demonstrate statistically significant weight loss in the largest study, leading to development termination in 2007

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