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dc.contributor.advisorLevy, Simonen_US
dc.contributor.authorKwak, Peteren_US
dc.date.accessioned2018-09-10T14:31:31Z
dc.date.available2018-09-10T14:31:31Z
dc.date.issued2018
dc.identifier.urihttps://hdl.handle.net/2144/31237
dc.description.abstractPermanent repair of the F9 gene is a significant goal to cure Hemophilia B disease. Advanced gene therapy using CRISPR/Cas9 system can increase circulation level of Factor IX proteins to a significant level without the need of demanding infusions of FIX concentrates. Induced pluripotent stem cells represent an ideal cell for gene therapy because patient-derived cells could be reprogrammed into iPSCs, genetically modified, selected, expanded and then induced to differentiate into fully functional hepatocytes in vitro. This study covered a portion of a 5-year project which ultimately aims at establishing therapeutic results in transgenic Hemophilia B mice by injecting genetically corrected iPSC-derived hepatocytes into the liver. The purpose of this thesis is to summarize what has been completed up to now: generation of the proper model of Hemophilia B human iPSCs using CRISPR/Cas9-mediated genome editing and differentiation of healthy and disease specific iPSCs into hepatocytes which will allow disease modelling to look for cell function, viability, homogeneity and drug screening. Further research will be done to effectively knock-in the F9 allele into liver safe harbor site of disease specific iPSCs, which will express FIX at a significant level to show therapeutic effects.en_US
dc.language.isoen_US
dc.subjectMedicineen_US
dc.subjectCRISPR/Cas9en_US
dc.subjectF9en_US
dc.subjectFactor IXen_US
dc.subjectHemophilia Ben_US
dc.subjectHepatocytesen_US
dc.subjectiPSCen_US
dc.titleGeneration of hemophilia B model hepatocyte derived from human iPSC via CRISPR/Cas9 mediated genome editingen_US
dc.typeThesis/Dissertationen_US
dc.date.updated2018-07-12T22:02:24Z
etd.degree.nameMaster of Scienceen_US
etd.degree.levelmastersen_US
etd.degree.disciplineMedical Sciencesen_US
etd.degree.grantorBoston Universityen_US


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