CRISPR/Cas9 Mediated Therapeutic Approach in Huntington's Disease

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dc.authoridAlkanli, Nevra/0000-0002-3745-8838
dc.authorwosidAlkanli, Nevra/D-4400-2019
dc.contributor.authorAlkanli, Suleyman Serdar
dc.contributor.authorAlkanli, Nevra
dc.contributor.authorAy, Arzu
dc.contributor.authorAlbeniz, Isil
dc.date.accessioned2024-06-12T11:03:24Z
dc.date.available2024-06-12T11:03:24Z
dc.date.issued2023
dc.departmentTrakya Üniversitesien_US
dc.description.abstractThe pathogenic mechanisms of these diseases must be well understood for the treatment of neurological disorders such as Huntington's disease. Huntington's Disease (HD), a dominant and neurodegenerative disease, is characterized by the CAG re-expansion that occurs in the gene encoding the polyglutamine-expanded mutant Huntingtin (mHTT) protein. Genome editing approaches include zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and Clustered Regularly Interspaced Short Palindromic Repeats/Caspase 9 (CRISPR/Cas9) systems. CRISPR/Cas9 technology allows effective gene editing in different cell types and organisms. Through these systems are created isogenic control of human origin induced pluripotent stem cells (iPSCs). In human and mouse models, HD-iPSC lines can be continuously corrected using these systems. HD-iPSCs can be corrected through the CRISPR/Cas9 system and the cut-and-paste mechanism using isogenic control iPSCs. This mechanism is a piggyBac transposon-based selection system that can effectively switch between vectors and chromosomes. In studies conducted, it has been determined that in neural cells derived from HD-iPSC, there are isogenic controls as corrected lines recovered from phenotypic abnormalities and gene expression changes. It has been determined that trinucleotide repeat disorders occurring in HD can be cured by single-guide RNA (sgRNA) and normal exogenous DNA restoration, known as the single guideline RNA specific to Cas9. The purpose of this review in addition to give general information about HD, a neurodegenerative disorder is to explained the role of CRISPR/Cas9 system with iPSCs in HD treatment.en_US
dc.identifier.doi10.1007/s12035-022-03150-5
dc.identifier.endpage1498en_US
dc.identifier.issn0893-7648
dc.identifier.issn1559-1182
dc.identifier.issue3en_US
dc.identifier.pmid36482283en_US
dc.identifier.scopus2-s2.0-85143655024en_US
dc.identifier.scopusqualityQ1en_US
dc.identifier.startpage1486en_US
dc.identifier.urihttps://doi.org/10.1007/s12035-022-03150-5
dc.identifier.urihttps://hdl.handle.net/20.500.14551/21645
dc.identifier.volume60en_US
dc.identifier.wosWOS:000895563500001en_US
dc.identifier.wosqualityQ2en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherSpringeren_US
dc.relation.ispartofMolecular Neurobiologyen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.subjectHuntington's Diseaseen_US
dc.subjectNeurodegenerative Disordersen_US
dc.subjectCRISPRen_US
dc.subjectCas9en_US
dc.subjectGenome Editing Systemsen_US
dc.subjectIpscen_US
dc.subjectIsogenic Cell Linesen_US
dc.subjectPluripotent Stem-Cellsen_US
dc.subjectControlled-Trialen_US
dc.subjectDouble-Blinden_US
dc.subjectGeneen_US
dc.subjectInhibitionen_US
dc.subjectMechanismsen_US
dc.subjectDiscoveryen_US
dc.subjectAciden_US
dc.titleCRISPR/Cas9 Mediated Therapeutic Approach in Huntington's Diseaseen_US
dc.typeArticleen_US

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