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A genetic disease prioritization platform for gene therapy studies: A wide-ranging review

dc.rights.licenseCC-BY-NC-ND
dc.contributor.advisorFuchs, Sabine
dc.contributor.authorSchürmann, Paul
dc.date.accessioned2023-07-22T00:02:01Z
dc.date.available2023-07-22T00:02:01Z
dc.date.issued2023
dc.identifier.urihttps://studenttheses.uu.nl/handle/20.500.12932/44270
dc.description.sponsorshipUtrecht University
dc.language.isoEN
dc.subjectThis review explores the challenge of treating over 10,000 global genetic disorders by targeting their root cause; DNA mutations. Using CRISPR technology as a basis for treating genetic diseases, we review publicly available databases and CRISPR gene editing prediction algorithms for their suitability to screen for diseases with the correct technical, medical and ethical properties that could be considered for gene correction therapy.
dc.titleA genetic disease prioritization platform for gene therapy studies: A wide-ranging review
dc.type.contentMaster Thesis
dc.rights.accessrightsOpen Access
dc.subject.courseuuDrug Innovation
dc.thesis.id19867


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