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CRISPR-based functional genomics in pluripotent stem cells

dc.contributor.authorZahedi, Setareh
dc.date.accessioned2025-12-09T14:51:49Z
dc.date.available2025-12-09T14:51:49Z
dc.date.issued2025-11-14
dc.description.abstractThe integration of CRISPR-based functional genomics with pluripotent stem cell (PSC) technologies has been recognized as a transformative approach for investigating gene function, modeling human disease, and advancing regenerative medicine. The aim of this review is to provide a comprehensive evaluation of recent developments in CRISPR-Cas platforms, including gene knockouts, base and prime editing, and CRISPR activation or interference (CRISPRa/i), as applied to PSC systems. Studies employing human PSCs, including embryonic stem cells and induced pluripotent stem cells, have been examined to summarize methodologies for genome-wide screening, lineage tracing, and therapeutic engineering. Advances in editing efficiency, delivery strategies, and genomic safety have been reported, while limitations persist in the form of off-target modifications, epigenetic variability, and cell-type-specific responses. Notable applications include the generation of immune-evasive PSC lines, the development of organoid models for physiological and pathological studies, and the implementation of phenotypic screening for disease-relevant traits. Collectively, these technological and methodological advancements have established functional genomics of PSC-CRISPRSPR as a versatile and powerful framework for elucidating fundamental aspects of human biology, dissecting complex traits, and accelerating the translation of discoveries from experimental research to clinical implementation.eng
dc.identifier.doi10.1007/s12015-025-11019-y
dc.identifier.eissn2629-3277
dc.identifier.issn2629-3269
dc.identifier.urihttp://hdl.handle.net/10400.1/27935
dc.language.isoeng
dc.peerreviewedyes
dc.publisherSpringer
dc.relation.ispartofStem Cell Reviews and Reports
dc.rights.uriN/A
dc.subjectCRISPR
dc.subjectCRISPR-Cas systems
dc.subjectPluripotent stem cells
dc.subjectEmbryonic stem cells
dc.subjectInduced pluripotent stem cells
dc.subjectFunctional genomics
dc.subjectGenome editing
dc.subjectOrganoids
dc.subjectRegenerative medicine
dc.titleCRISPR-based functional genomics in pluripotent stem cellseng
dc.typejournal article
dspace.entity.typePublication
oaire.citation.titleStem Cell Reviews and Reports
oaire.versionhttp://purl.org/coar/version/c_970fb48d4fbd8a85

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