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Chapters
- 00:05Title
- 00:35Site-directed Mutagenesis
- 01:45Transformation and Screening for the Desired Clone
- 02:35pcDNA3.1/GLA and pcDNA3.1/GAA Plasmid Transfection and Treatment of HEK293H
- 04:00Cell Harvest
- 04:43Homogenization by Freezing and Thawing and BCA Assay
- 06:17Enzyme Activity Measurement with Artificial 4-Methylumbeliferyl Substrate
- 08:19Results: Enzyme Activity Measurement Mutagenized 3 α-galactosidase and 3 Acid α-glucosidase
- 09:35Conclusion
C'è una domanda per fare pre-clinica test per una nuova classe di farmaci "orfani" chiamato chaperoni farmacologici riproducibile, veloce ed efficiente. Abbiamo sviluppato un test cellulari semplice, altamente standardizzate e versatile cultura alla schermata per i pazienti eleggibili come pure droghe romanzo chaperoni farmacologici.
Tags
In VitroEnzyme MeasurementPharmacological ChaperoneFabry DiseasePompe DiseasePhenotypic AssessmentAllelic VarianceLysosomal Storage DiseasesPrognostic OutcomesTherapeutic DecisionsSite-directed MutagenesisGLA GeneGAA GenePrimer DesignPCRDpn1E.coliPlasmid PurificationSpectrophotometerHEK293H CellsCell Transfection
